disadvantages of gene therapy essay

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What Is Gene Therapy?

A New Type of Therapy for Genetic Diseases

• Why It's Done
• What to Expect

Frequently Asked Questions

Gene therapy is a type of treatment being developed to fight diseases that are caused by genetic defects. This is a relatively new medical intervention that is mainly in the experimental phase, including human trials and animal trials, for the treatment of some conditions, such as cystic fibrosis .

Gene therapy aims to change the unhealthy proteins that are produced as a result of disease-causing genes.

KATERYNA KON / SCIENCE PHOTO LIBRARY / Getty Images

What Is Gene Therapy? 

Some diseases are caused by a known genetic defect or a gene mutation. This means that there is a hereditary or acquired error in the DNA molecule that codes for producing a specific protein in the body. The altered protein doesn’t function as it should, resulting in a disease. 

The idea behind gene therapy is to direct the body to produce healthy proteins that do not cause disease.

This therapy involves the delivery of DNA or RNA. The RNA molecule is an intermediate molecule that is formed in the process of protein production. The genetic defect for some diseases has been identified, but many genetic mutations haven’t been identified (they might be in the future). 

Research is ongoing into ways to correct genetic defects that have been associated with certain diseases. There are different types and methods of gene therapy that are being investigated. 

Types of Gene Therapy 

Genetic mutations can be hereditary, which means that they are inherited from parents. Genetic defects can also be acquired, sometimes due to environmental factors, like smoking. 

Gene therapy is being evaluated as a potential treatment for both types of mutations. There are several ways of delivering the corrected DNA or RNA into a person’s body.

Most cells in your body are somatic cells. The only cells that are not somatic cells are the germline cells, which create the egg and sperm cells that can produce offspring.

Somatic gene therapy : Somatic gene therapy aims to correct a defect in the DNA of a somatic cell or to provide an RNA molecule to treat or prevent a genetic disease in the person who is undergoing the therapy. This treatment may be used if you have an inherited mutation or if the mutation developed due to environmental factors.

Germline gene therapy : Germline gene therapy aims to correct a defect in an egg or a sperm cell to prevent a hereditary disease from eventually affecting future offspring.

Bone Marrow 

Sometimes a person’s own cells can be removed from the bone marrow , genetically modified in a laboratory, and then reinserted into the body.

Viral Vector 

A viral vector is a virus that has been altered so that it will not cause a viral infection. It is then infused with the correct DNA or RNA sequence. The viral vector containing the correct gene may be injected into a person for delivery of gene therapy.

Stem Cells 

Stem cells are immature cells that have the potential to develop into different types of cells. Sometimes stem cells that have been genetically modified are transplanted into a person’s body to replace the defective cells as a way to treat disease. 

This technique uses a lipid (fat) to deliver the genetic DNA or RNA material. 

Why Is Gene Therapy Done? 

Some gene mutations direct the body to make disease-causing proteins. And some genetic mutations are not functional—they cause disease because the body lacks the healthy proteins that should be normally produced by the gene.

Gene therapy aims to direct the body to produce healthy proteins or to inhibit the production of defective proteins. This depends on the type of mutation that is causing the disease. 

Gene Augmentation Therapy: Replacing Mutated Genes

With gene augmentation, the goal is to help the body make a healthy protein.

Sometimes the DNA molecule can have a gene inserted into it. This is intended to permanently alter the DNA so that the body can make new cells with the correct DNA code. The new cells will then also make healthy products. 

Some research using gene augmentation therapy involves the insertion of a healthy DNA molecule or an RNA sequence into a cell, but not into the DNA of the recipient. This has been shown in experimental studies to trigger the production of healthy proteins, but future copies of the cell are not expected to contain the healthy gene. 

Gene Inhibition Therapy: Inactivating Mutated Genes 

Sometimes gene therapy aims to cancel out the activity of a mutated gene to prevent the production of a disease-causing protein. This is done by the insertion of a non-mutated gene DNA sequence into a DNA molecule. 

Making Diseases Cells Apparent to the Immune System 

Another type of gene therapy involves the body’s immune system . An example of this therapy is the use of checkpoint inhibitors. With this therapy, the immune system is modified to recognize material in the body that is produced by the mutated genes in order to destroy them and to prevent the illnesses they cause. 

Risks of Gene Therapy 

There are some known risks of gene therapy. So far, the most common problem associated with gene therapy is a lack of effectiveness. However, there are also adverse effects that may occur. 

Unwanted Immune System Reaction 

Gene therapy that involves the immune system may cause excess immune reactivity to healthy cells that resemble the disease cells, potentially causing damage to healthy cells.   

Wrong Target Cell

Potentially, the immune reaction that is mediated by gene therapy can affect the wrong cell type, instead of the intended target cells. 

Infection Caused by Viral Vector 

When a viral vector is used, there may be a risk that the virus could cause an infection. Depending on the primary disease that is being treated, a person receiving gene therapy may have a weak immune system and, therefore, could have difficulty fighting the virus. 

Possible Tumor

A new DNA sequence that’s inserted into a person’s genes could potentially lead to a mutation that could cause cancer to form. 

What to Expect From Gene Therapy 

If you are considering gene therapy, you will go through a process of diagnosis, treatment, and medical surveillance to assess the effects. 

This step will determine whether you have a medical condition that can be treated with gene therapy. This means that you would have a blood sample sent to a laboratory to identify treatable gene mutations that are associated with your medical condition.

Examples of conditions that may be treatable with gene therapy include:

• Cystic fibrosis : An inherited disorder in which thick mucus is produced, clogging the airways and blocking the secretion of digestive enzymes
• Sickle cell disease : An inherited disorder that results in abnormal hemoglobin production (the protein that carries oxygen in the red blood cells)
• Leber's hereditary optic neuropathy (LHON) : An inherited disorder that causes the death of cells in the optic nerve , resulting in damage to central vision
• Inherited or acquired retinal disease : Conditions that damage to the retina , the light-sensing layer in the back of the eye
• WW domain-containing oxidoreductase (WWOX) epileptic encephalopathy syndrome : A genetic condition resulting in severe epilepsy, developmental delays, and early death
• Spinocerebellar ataxia and autosomal recessive 12 (SCAR12) : An inherited disorder resulting in seizures in infancy, developmental delays, and inability to coordinate movement
• Cancer : Many types of cancer

Your treatment may involve collection of your cells and delivery of the genes into your cells with a viral vector or liposome. The modified cells will be restored to your body after the treatment. 

Surveillance

The effects of your treatment will be assessed, and you will be monitored for adverse events (side effects). If this occurs, you may be treated again. 

Clinical Trials

You can find clinical trials for gene therapy by talking to your doctor or by searching for organizations that support your medical condition, such as the Cystic Fibrosis Foundation.

Gene therapy is a relatively new treatment designed to alleviate disease by modifying defective genes or altering the production of proteins by faulty genes. There are several ways that healthy genes can be inserted into the body, such as inside a deactivated virus or inside a fat particle.

Sometimes immature and healthy cells are transplanted to replace cells that have a disease-causing mutation. This type of therapy can cause side effects, and there is also a risk that it might not work. 

A Word From Verywell 

If you have a genetic disease with a known and identified gene mutation, you might be a candidate for gene therapy treatment in a clinical trial. This type of treatment is not a standard therapy, and you would need to be monitored closely so that you and your doctors would know if the therapy is working and whether you are having any side effects.

You can talk to your doctor about gene therapy. This treatment is not widespread, so there is a possibility that you may need to travel to be able to participate in a clinical trial if there is not a research study near you. 

This therapy is considered safe, but there are risks and side effects. You may have an opportunity to participate in a clinical trial, and side effects and adverse effects would be monitored. 

One example of this therapy is the use of a deactivated virus to insert a portion of a DNA molecule into the body’s cells so that the healthy DNA sequence can provide a blueprint for healthy proteins. 

The main goal of gene therapy is to provide DNA or RNA to code for healthy proteins so the body will not be affected by a genetic disease. 

Cystic Fibrosis Foundation. Gene therapy for cystic fibrosis .

Food and Drug Administration. What is gene therapy?

Jiang J, Sun G, Miao Q, Li B, Wang D, Yuan J, Chen C. Observation of peripapillary choroidal vascularity in natural disease course and after gene therapy for Leber's hereditary optic neuropathy. Front Med (Lausanne) . 2021;8:770069. doi:10.3389/fmed.2021.770069

Repudi S, Kustanovich I, Abu-Swai S, Stern S, Aqeilan RI. Neonatal neuronal WWOX gene therapy rescues Wwox null phenotypes . EMBO Mol Med. 2021;13(12):e14599. doi:10.15252/emmm.202114599

Tan TE, Fenner BJ, Barathi VA, Tun SBB, Wey YS, Tsai ASH, Su X, Lee SY, Cheung CMG, Wong TY, Mehta JS, Teo KYC. Gene-based therapeutics for acquired retinal disease: Opportunities and progress . Front Genet. 2021;12:795010. doi:10.3389/fgene.2021.795010

Lu Z, Chen H, Jiao X, et. al. Germline HLA-B evolutionary divergence influences the efficacy of immune checkpoint blockade therapy in gastrointestinal cancer . Genome Med . 2021;13(1):175. doi:10.1186/s13073-021-00997-6

By Heidi Moawad, MD Dr. Moawad is a neurologist and expert in brain health. She regularly writes and edits health content for medical books and publications.

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• Benefits and Risks
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MORE INFORMATION

Genetic Therapies Benefits and Risks

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In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders, such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. They also may be used to treat cancers or infections, including HIV.

Genetic therapies that are currently approved by the FDA are available for people who have  Leber congenital amaurosis , a rare inherited condition that leads to blindness.  CAR T-cell therapy  is FDA approved for people who have blood cancers, such as  acute lymphoblastic leukemia (ALL)  and diffuse large B-cell lymphoma.

Genetic therapies hold promise to treat many diseases, but they are still new approaches to treatment and may have risks. Potential risks could include certain types of cancer, allergic reactions, or damage to organs or tissues if an injection is involved.

Recent advances have made genetic therapies much safer. Better safety has resulted in the FDA approving some gene transfer therapies for clinical use in the United States. There have been a few clinical studies on genome editing, but the approach is much newer than gene transfer. Researchers are still studying the risks.

The National Institutes of Health, which includes the NHLBI, does not perform or fund studies on genome editing targeting sperm, eggs, or embryos in humans. These changes would be passed on to the patient’s children and could have unanticipated effects.

12 Pros and Cons of Gene Therapy

Gene therapy is the process of transplanting genes that have developed normally in place of genes that may be missing or have developed abnormally to correct a genetic disorder. It is a technique that is still in its experimental stages, but has shown some promising results for some individuals. More than 2,300 clinical trials involving gene therapy have been conducted since 1989.

The advantage of gene therapy is that it provides an opportunity for affected individuals to lead a life that is “normal.” Some gene therapies may even offer the chance to survive because the genetic disorder is diagnosed as being terminal without an intervention.

The disadvantage of gene therapy is that, like any other medical procedure, it may not work. The experimental nature of these therapies means that the costs can be high to have them performed and many health insurance policies will not cover it because of the potential of failure.

Here are some additional pros and cons of gene therapy to think about.

What Are the Pros of Gene Therapy?

1. It offers hope. Even with extensive screening in place for parents, there are numerous births that experience defects and genetic diseases. In the United States, 1 in every 33 babies experiences a birth defect of some sort. That’s about 3% of all births. Birth defects are the leading cause of infant death in the US, accounting for 1 in 5 fatalities. With gene therapy, some of these defects could be corrected to reduce these fatalities.

2. Genetic disorders can be treated. Rare diseases affect about 10% of the general population. More than 30 million people in the United States are affected. There are over 7,000 distinct diseases that exist and about 80% of those diseases are caused by faulty genetics. With gene therapy, normal cells could replace the faulty cells and provide a legitimate treatment for the affected individual.

3. It may treat more than just disease. Gene therapy techniques could provide individuals suffering from infertility with the opportunity to start their own biological families. Using a modified gene therapy method called “CRISPR,” successful alterations to fertility in mice have occurred. This creates the potential of creating a similar effect in humans one day.

4. It would create a new field of medicine. Gene therapy offers the chance to treat numerous diseases and disorders that currently do not have a treatment protocol. Even age-related disorders that may have genetic foundations, such as Alzheimer’s disease, could be treated with the medicines and protocols that come from genetic therapeutic research.

5. Gene therapies aren’t limited to humans. When gene therapies are applied to veterinary science, it becomes possible to extend the lifespan of animals. We could treat genetic conditions in animals to prevent loss. This would stabilize our animal protein food chain when applied to livestock. It could be applied to plants so that they can naturally withstand disease without additional DNA and genes added to them. The opportunities to help life are virtually endless, in whatever form it may take.

6. Gene therapy is based on technology. Because gene therapies are technologically-based, their overall cost will drop as new methods and advancements enter the medical field. Initial treatments may be expensive, but the future of treatment in the coming generations may not be at all. As more research in this field occurs, prices will drop. We’ve already seen this with penicillin. When it was first introduced, the price was $20 per 100,000 units. With a typical dose being 4 million units, that’s the equivalent price of $70,000 per treatment. Today, the price of penicillin is just pennies per dose in many circumstances.

What Are the Cons of Gene Therapy?

1. It is a costly treatment option. Even if gene therapy becomes an accepted form of treatment for the diseases that affect humanity, the cost of administering them could create socioeconomic classes that are based on wealth and health. Individuals could be tested at birth for genetic disorders and then classified into risk pools that may limit their access to care or provide them at an extreme cost. The first approved gene therapy treatment in Europe, Alipogene tiparvovec, is $1 million per treatment. Only one person had ever been treated with the drug since 2016.

2. Nature is adaptable. As we have seen with the growing resistance to antibiotics, nature can readily adapt to changes that occur. Gene therapies may be useful now, but additional changes to genetic profiles could create unforeseen disorders in the future. There is no guarantee that the future potential of gene therapy can live up to its current potential to treat specific disorders. By manipulating genes, we could be creating new disorders for future generations without realizing it.

3. It may unlock unethical forms of science. When humanity has the knowledge to manipulate a genetic profile, the science of eugenics becomes possible. It may create a future where children have their genetic profiles altered in vitro so that a specific result is created. This would create different “standards” in humanity, creating a different class of person who has been “perfected.” It would also highly the wealth gaps that exist in many societies because this service would only be available to those who could afford it.

4. Gene therapies have been stuck in trials for a generation for a good reason. Many of the gene therapies that currently exist have been proven to be mostly ineffective. Conditions that are treated by a gene therapy improve for a short time, but then revert to the state they were before treatment began. For gene therapies that are successful, ongoing treatments or tissue donations, such as bone marrow, may be required.

5. It may encourage gene doping. Although gene doping is not known to presently exist, it is a process that could equalize athletics or educational opportunities if equal access to the technology is given. If a person succeeds because of gene therapy when they may not have the same levels of success without it is a subject of ethical concern, especially when considering athletic competition.

6. It can provide a false hope. Gene therapies have saved children from leukemia. In the case of Charlie Gard and others in a similar situation, the promise of gene therapy may provide a false hope of survival. There are times when saving someone may create an inferior quality of life. Is the person being saved for their benefit… or the benefit of their loved ones?

The pros and cons of gene therapy show us that there are some uncomfortable questions that need to be answered as this technology progresses. The hope it provides is extraordinary. There may also be unforeseen side effects that could cause more harm than good as we work to treat some of the most devastating conditions that humans experience right now.

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• What are the ethical issues surrounding gene therapy?

Because gene therapy involves making changes to the body’s basic building blocks (DNA), it raises many unique ethical concerns. The ethical questions surrounding gene therapy and genome editing include:

How can “good” and “bad” uses of these technologies be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current research on gene therapy treatment has focused on targeting body (somatic) cells such as bone marrow or blood cells. This type of genetic alteration cannot be passed to a person’s children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the genetic changes to be passed to future generations. This approach is known as germline gene therapy.

The idea of these germline alterations is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

Topics in the Gene Therapy and Other Medical Advances chapter

• What is gene therapy?
• How does gene therapy work?
• Is gene therapy safe?
• Is gene therapy available to treat my disorder?
• What are CAR T cell therapy, RNA therapy, and other genetic therapies?
• What are mRNA vaccines and how do they work?

Other chapters in Help Me Understand Genetics

Genetics Home Reference has merged with MedlinePlus. Genetics Home Reference content now can be found in the "Genetics" section of MedlinePlus. Learn more

The information on this site should not be used as a substitute for professional medical care or advice. Contact a health care provider if you have questions about your health.

14 Advantages and Disadvantages of Gene Therapy

Gene therapy is a medically-based practice that uses normalized genetics to replace genes which are either not present or abnormal for some individuals. Doctors would take the specific gene sequences that need adjustment, and then insert them into the cellular information of the patient in various ways. Most forms of gene therapy are still in the clinical research stage, but there have been stories of encouraging results.

Several inherited immune deficiencies are being treated successfully right now with gene therapy. When the blood stem cells are removed from patients, retroviruses then deliver working copies of the defective genes to the body.

For the gene therapy options which have been approved for use, there are many success stories to consider. Sebastian Misztal is one such story. He was a patient in a hemophilia gene therapy trial in 2011. After receiving the therapy, Misztal no longer experiences episodes of spontaneous bleeding.

Roughly 70% of the currently active gene therapy clinical trials are based in the United States. Europe approved their first treatment in this area in 2012. These are the pros and cons of this scientific approach to consider.

List of the Pros of Gene Therapy

1. Gene therapy provides hope for those who may not have had any in the past. About 3% of American children are born with a genetic condition which requires gene therapy as a way to treat the issue. At this time, the diseases and disorders which are present in this population will take the life of the child before there is an opportunity to correct the condition. Birth defects are the leading cause of newborn death in the United States, with as many as 1 in 5 children suffering from them. Advances in gene therapy could help to correct these issues instead of forcing parents into a heartbreaking scenario.

2. Gene therapy could change the perspectives that people have about disease. Roughly 10% of all Americans are affected by a rare disease or condition on any given day. Approximately 33 million people are suffering from a disability that is directly attributed to their genetic profile. The promise of gene therapy is that it can reduce or eliminate the pain and discomfort that these abnormalities cause. 80% of the diseases that we know impact human health in negative ways have a genetic foundation. If we can replace the cells or chromosomes that are at-fault, then it becomes possible to offer relief.

3. Gene therapy could offer the potential of new discoveries. Our world is a better place when there is an emphasis on diversity. When we have effective gene therapy treatments that can save lives or prolong them, then we are adding strength to our existence. There will be more opportunities to research, new ideas that could lead to critical discoveries, and relationships that can lead to future generations that experience these benefits as well. There will always be a segment of society that looks at gene therapy as a way to “play God.” The reality of this medical treatment is that it can help people continue to live a life that they love.

4. Gene therapy could be used in different ways to improve life. Right now, the focus of gene therapy research is to provide solutions for people who are suffering from specific illnesses or diseases. When we begin to experience successes in this field, then the information we learn can apply to other treatment areas as well. Gene therapy could be useful in the treatment of infertility issues. The processes involved may help people struggling with vision or hearing issues. Even if the only thing that we can do with this science is to relieve chronic pain, that would be tremendously beneficial for the futures of many people.

5. Gene therapy does not just apply to human treatment options. When we discuss the pros and cons of gene therapy, it is essential to remember that the benefits we can experience as humans apply to other forms of life as well. The technologies we create from this research could help us to grow crops that adapt more effectively to changing climate conditions. We could use this information to correct the various genetic conditions that we know about in the animal kingdom. This data could help us to grow healthier foods, increase the shelf life of harvests, or produce more items in our overall yields.

6. Gene therapy allows us to use technology to improve the quality of life for people. Many of our medical discoveries rely on technological processes that we apply to natural items. Even some of the most critical advancements of our era, such as the development of a polio vaccine by Dr. Jonas Salk (and the work of many others) relied on the use of inactivated virus materials to create the first usable product. Gene therapy would become one of the first treatment options for doctors that was purely technological. That means our opportunity to develop new resources from it are virtually unlimited.

7. Gene therapy allows us to treat the “untreatable” diseases. Gene therapy is potential miracle worker when we start to look at its full potential for humanity. It offers us the opportunity to eliminate, and then prevent hereditary diseases like hemophilia and cystic fibrosis. The technologies behind this treatment option could provide us with a possible cure for heart disease. Potential medical options include cancer and AIDS cures. Even if there is a fair amount of risk involved when treating these health issues, there are a lot of patients who don’t have much to lose. Gene therapy opens a door that we once thought was permanently locked.

List of the Cons of Gene Therapy

1. Gene therapy does not have a reliable delivery method. Retrovirus delivery systems are the most common way for gene therapies to be delivered to patients. The problem with this option is that the enzyme used to encourage the transfer of genetic data can be eliminated by the immune system before it has the chance to work. There could be issues with cell division or replication that limit the effectiveness of the treatment.

When there is a noticeable change to the cell, the body might attack itself without the presence of an immunosuppressant. Until we can remove and replace genetic data with more reliability, the success stories for gene therapy will always be hit or miss.

2. Gene therapy is an expensive procedure. There are several gene therapy options which are available right now, but they come at a steep price. If you use Luxturna to treat both eyes as a way to treat blindness, then the final cost could be more than $1 million. Even the “affordable” options in this field start at $200,000 per treatment. That’s why many patients weight for clinical trials to begin, and then apply for a spot in one to receive the help they need. Most healthcare insurance plans will not cover the cost of these procedures because of their uncertainty.

3. Gene therapy requires ongoing treatment s to be effective. Many people have found that the benefits of their gene therapy treatments began to wear off as soon as they were no longer taking their medicine or visiting their doctor for treatments. It can be a lifelong course that someone must follow to reduce or eliminate the genetic issues that hold back their health. Unless you can keep taking the products which are often priced above $100,000 per treatment, then you will experience a reversal in your condition.

4. Gene therapy may not be able to adapt to a changing world. It has taken less than a century for prescription-grade antibiotics to no longer be as effective for the treatment of bacterial infections as it once was. Antibiotic resistance can impact anyone at any age, and in any country. Sometimes it occurs naturally, but the most common reason for this issue is that antibiotic misuse has led to a growing number of infections, including pneumonia, tuberculosis, and salmonellosis being more challenging to treat because the medicine is not as effective against the bacteria.

This issue could occur with gene therapies too. We have already seen people begin to have their progress reverse itself when they stop following their treatment plan. Over the next couple of generations, the body could start resisting this option too.

5. Gene therapy might only delay the inevitable. Jolee Mohr was lying in a Chicago hospital, her body swollen by internal bleeding and organ failure. The sight was so difficult that her husband decided not to bring their 5-year-old daughter into the room to say goodbye. Although there was no evidence to suggest a link, Jolee had taken an experimental treatment for rheumatoid arthritis. She was only 36 years old.

The National Institutes of Health approved the first human gene transfer study in 1989. Through 2006, there were 800 gene therapy studies that involved 5,000 patients. In those 17 years, the total number of approved therapies was zero. The only success story was a cure for the “bubble boy” disease that also caused leukemia thanks to the virus that delivered the treatment.

And Jolee wasn’t the only story. A teenager named Jesse Gelsinger also died because of treatments offered inn a clinical research study. We must remember that there are sad stories to tell in addition to the happy ones when evaluating this treatment option.

6. Gene therapy will shift society toward new polarization. The United States is already highly polarized from a political perspective If gene therapies are approved for widespread use, then it may create another layer of separation from a medical perspective. Although most people can get behind the idea of creating a cure for cancer, birth defects, or chromosomal disorders, the processes used could also create designer genetics that promise a specific outcome. Should we pursue a scientific field that could help our children become smarter, faster, or better looking?

7. Gene therapy could change the way we think about competition. Although the discussion of “designer babies” often involves looks, the science behind gene therapy could also encourage specific traits to develop in children. Parents with wealth could work with their doctors to support a healthier muscle mass, faster fat burning capabilities, or an adaptive body frame that allows for greater flexibility in sports. People could design an outcome where results could follow a curve where outcomes could be planned for years in advance. This process would result in another layer of socioeconomic separation that would likely lead to even more polarization.

The pros and cons of gene therapy still require a lot of soul searching, even though we are 30 years and counting into this field of research. We are beginning to see some successes, but it has also come at the expense of some high-profile failures. Only time will tell if we can put this information to good use for the betterment of humanity. Until then, we must continue searching to find more solutions to the significant health issues our race faces each day.

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Harvard researchers, others share their views on key issues in the field

Medicine is at a turning point, on the cusp of major change as disruptive technologies such as gene, RNA, and cell therapies enable scientists to approach diseases in new ways. The swiftness of this change is being driven by innovations such as CRISPR gene editing , which makes it possible to correct errors in DNA with relative ease.

Progress in this field has been so rapid that the dialogue around potential ethical, societal, and safety issues is scrambling to catch up.

This disconnect was brought into stark relief at the Second International Summit on Human Genome Editing , held in Hong Kong in November, when exciting updates about emerging therapies were eclipsed by a disturbing announcement. He Jiankui, a Chinese researcher, claimed that he had edited the genes of two human embryos, and that they had been brought to term.

There was immediate outcry from scientists across the world, and He was subjected to intense social pressure, including the removal of his affiliations, for having allegedly disregarded ethical norms and his patients’ safety.

Yet as I. Glenn Cohen, faculty director of the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School, has said, gene editing comes in many varieties, with many consequences. Any deep ethical discussion needs to take into account those distinctions.

Human genome editing: somatic vs. germline

The germline editing He claimed to have carried out is quite different from the somatic gene therapies that are currently changing the frontiers of medicine. While somatic gene editing affects only the patient being treated (and only some of his or her cells), germline editing affects all cells in an organism, including eggs and sperm, and so is passed on to future generations. The possible consequences of that are difficult to predict.

Somatic gene therapies involve modifying a patient’s DNA to treat or cure a disease caused by a genetic mutation. In one clinical trial, for example, scientists take blood stem cells from a patient, use CRISPR techniques to correct the genetic mutation causing them to produce defective blood cells, then infuse the “corrected” cells back into the patient, where they produce healthy hemoglobin. The treatment changes the patient’s blood cells, but not his or her sperm or eggs.

Germline human genome editing, on the other hand, alters the genome of a human embryo at its earliest stages. This may affect every cell, which means it has an impact not only on the person who may result, but possibly on his or her descendants. There are, therefore, substantial restrictions on its use.

Germline editing in a dish can help researchers figure out what the health benefits could be, and how to reduce risks. Those include targeting the wrong gene; off-target impacts, in which editing a gene might fix one problem but cause another; and mosaicism, in which only some copies of the gene are altered. For these and other reasons, the scientific community approaches germline editing with caution, and the U.S. and many other countries have substantial policy and regulatory restrictions on using germline human genome editing in people.

But many scientific leaders are asking: When the benefits are believed to outweigh the risks, and dangers can be avoided, should science consider moving forward with germline genome editing to improve human health? If the answer is yes, how can researchers do so responsibly?

CRISPR pioneer Feng Zhang of the Broad Institute of Harvard and MIT responded immediately to He’s November announcement by calling for a moratorium on implanting edited embryos in humans. Later, at a public event on “Altering the Human Genome” at the Belfer Center at Harvard Kennedy School (HKS), he explained why he felt it was important to wait:

“The moratorium is a pause. Society needs to figure out if we all want to do this, if this is good for society, and that takes time. If we do, we need to have guidelines first so that the people who do this work can proceed in a responsible way, with the right oversight and quality controls.”

Professors at the University’s schools of medicine, law, business, and government saw He’s announcement as a turning point in the discussion about heritable gene therapies and shared their perspectives on the future of this technology with the Gazette.

Here are their thoughts, issue by issue:

Aside from the safety risks, human genome editing poses some hefty ethical questions. For families who have watched their children suffer from devastating genetic diseases, the technology offers the hope of editing cruel mutations out of the gene pool. For those living in poverty, it is yet another way for the privileged to vault ahead. One open question is where to draw the line between disease treatment and enhancement, and how to enforce it, considering differing attitudes toward conditions such as deafness.

Robert Truog , director of the Center for Bioethics at Harvard Medical School (HMS), provided context:

“This question is not as new as it seems. Evolution progresses by random mutations in the genome, which dwarf what can be done artificially with CRISPR. These random mutations often cause serious problems, and people are born with serious defects. In addition, we have been manipulating our environment in so many ways and exposing ourselves to a lot of chemicals that cause unknown changes to our genome. If we are concerned about making precise interventions to cure disease, we should also be interested in that.

“To me, the conversation around Dr. He is not about the fundamental merits of germline gene editing, which in the long run will almost certainly be highly beneficial. Instead, it’s about the oversight of science. The concern is that with technologies that are relatively easy to use, like CRISPR, how does the scientific community regulate itself? If there’s a silver lining to this cloud, I think it is that the scientific community did pull together to be critical of this work, and took the responsibility seriously to use the tools available to them to regulate themselves.”

When asked what the implications of He’s announcement are for the emerging field of precision medicine, Richard Hamermesh, faculty co-chair of the Harvard Business School/Kraft Precision Medicine Accelerator, said:

“Before we start working on embryos, we have a long way to go, and civilization has to think long and hard about it. There’s no question that gene editing technologies are potentially transformative and are the ultimate precision medicine. If you could precisely correct or delete genes that are causing problems — mutating or aberrant genes — that is the ultimate in precision. It would be so transformative for people with diseases caused by a single gene mutation, like sickle cell anemia and cystic fibrosis. Developing safe, effective ways to use gene editing to treat people with serious diseases with no known cures has so much potential to relieve suffering that it is hard to see how anyone could be against it.

“There is also commercial potential and that will drive it forward. A lot of companies are getting venture funding for interesting gene therapies, but they’re all going after tough medical conditions where there is an unmet need — [where] nothing is working — and they’re trying to find gene therapies to cure those diseases. Why should we stop trying to find cures?

“But anything where you’re going to be changing human embryos, it’s going to take a long time for us to figure out what is appropriate and what isn’t. That has to be done with great care in terms of ethics.”

George Q. Daley  is dean of HMS, the Caroline Shields Walker Professor of Medicine, and a leader in stem cell science and cancer biology. As a spokesperson for the organizing committee of the Second International Summit on Human Genome Editing, he responded swiftly to He’s announcement in Hong Kong. Echoing those remarks, he said:

“It’s time to formulate what a clinical path to translation might look like so that we can talk about it. That does not mean that we’re ready to go into the clinic — we are not. We need to specify what the hurdles would be if one were to move forward responsibly and ethically. If you can’t surmount those hurdles, you don’t move forward.

“There are stark distinctions between editing genes in an embryo to prevent a baby from being born with sickle cell anemia and editing genes to alter the appearance or intelligence of future generations. There is a whole spectrum of considerations to be debated. The prospect includes an ultimate decision that we not go forward, that we decide that the benefits do not outweigh the costs.”

Asked how to prevent experiments like He’s while preserving academic freedom, Daley replied:

“For the past 15 years, I have been involved in efforts to establish international standards of professional conduct for stem cell research and its clinical translation, knowing full well that there could be — and has been — a growing number of independent practitioners directly marketing unproven interventions to vulnerable patients through the internet. We advocated so strongly for professional standards in an attempt to ward off the risks of an unregulated industry. Though imperfect, our efforts to encourage a common set of professional practices have been influential.

“You can’t control rogue scientists in any field. But with strongly defined guidelines for responsible professional conduct in place, such ethical violations like those of Dr. He should remain a backwater, because most practitioners will adhere to generally accepted norms. Scientists have a responsibility to come together to articulate professional standards and live by them. One has to raise the bar very high to define what the standards of safety and efficacy are, and what kind of oversight and independent judgment would be required for any approval.

“We have called for an ongoing international forum on human genome editing, and that could take many shapes. We’ve suggested that the national academies of more countries come together — the National Academy of Sciences in the U.S. and the Royal Society in the U.K. are very active here — because these are the groups most likely to have the expertise to convene these kinds of discussions and keep them going.”

Cohen , speaking to the legal consequences of germline human genome editing, said:

“I think we should slow down in our reaction to this case. It is not clear that the U.S. needs to react to Dr. He’s announcement with regulation. The FDA [Food and Drug Administration] already has a strong policy on germline gene editing in place. A rider in the Consolidated Appropriations Act of 2016 — since renewed — would have blocked the very same clinical application of human germline editing He announced, had it been attempted in the U.S.

“The scientific community has responded in the way I’d have liked it to. There is a difference between ‘governance’ and ‘self-governance.’ Where government uses law, the scientific community uses peer review, public censure, promotions, university affiliations, and funding to regulate themselves. In China, in Dr. He’s case, you have someone who’s (allegedly) broken national law and scientific conventions. That doesn’t mean you should halt research being done by everyone who’s law-abiding.

“Public policy or ethical discussion that’s divorced from how science is progressing is problematic. You need to bring everyone together to have robust discussions. I’m optimistic that this is happening, and has happened. It’s very hard to deal with a transnational problem with national legislation, but it would be great to reach international consensus on this subject. These efforts might not succeed, but ultimately they are worth pursuing.”

Professor Kevin Eggan of Harvard’s Department of Stem Cell and Regenerative Biology said, “The question we should focus on is: Will this be safe and help the health of a child? Can we demonstrate that we can fix a mutation that will cause a terrible health problem, accurately and without the risk of harming their potential child? If the answer is yes, then I believe germline human genome editing is likely to gain acceptance in time.

“There could be situations where it could help a couple, but the risks of something going wrong are real. But at this point, it would be impossible to make a risk-benefit calculation in a responsible manner for that couple. Before we could ever move toward the clinic, the scientific community must come to a consensus on how to measure success, and how to measure off-target effects in animal models.

“Even as recently as this past spring and fall, the results of animal studies using CRISPR — the same techniques Dr. He claimed to have used — generated a lot of confusion. There is disagreement about both the quality of the data and how to interpret it. Until we can come to agreement about what the results of animal experiments mean, how could we possibly move forward with people?

“As happened in England with mitochondrial replacement therapy, we should be able to come to both a scientific and a societal consensus of when and how this approach should be used. That’s missing.”

According to Catherine Racowsky, professor of obstetrics, gynecology and reproductive biology at Brigham and Women’s Hospital, constraints on the use of embryos in federally funded research pose barriers to studying the risks and benefits of germline editing in humans. She added:

“Until the work is done, carefully and with tight oversight, to understand any off-target effects of replacing or removing a particular gene, it is inappropriate to apply the technology in the clinical field. My understanding of Dr. He’s case is that there wasn’t a known condition in these embryos, and by editing the genes involved with HIV infection, he could also have increased the risks of susceptibility to influenza and West Nile viruses.

“We need a sound oversight framework, and it needs to be established globally. This is a technology that holds enormous promise, and it is likely to be applied to the embryo, but it should only be applied for clinical purposes after the right work has been done. That means we must have consensus on what applications are acceptable, that we have appropriate regulatory oversight, and, perhaps most importantly, that it is safe. The only way we’re going to be able to determine that these standards are met is to proceed cautiously, with reassessments of the societal and health benefits and the risks.”

Asked about public dialogue around germline human genome editing, George Church , Robert Winthrop Professor of Genetics at HMS, said:

“With in vitro  fertilization (IVF), ‘test tube babies’ was an intentionally scary term. But after Louise Brown, the first IVF baby, was born healthy 40 years ago, attitudes changed radically. Ethics flipped 180 degrees, from it being a horrifying idea to being unacceptable to prevent parents from having children by this new method. If these edited twins are proven healthy, very different discussions will arise. For example, is a rate of 900,000 deaths from HIV infection per year a greater risk than West Nile virus, or influenza? How effective is each vaccine?”

Science, technology, and society

Sheila Jasanoff , founding director of the Science, Technology, and Society program at HKS, has been calling for a “global observatory” on gene editing, an international network of scholars and organizations dedicated to promoting exchange across disciplinary and cultural divides. She said:

“The notion that the only thing we should care about is the risk to individuals is very American. So far, the debate has been fixated on potential physical harm to individuals, and not anything else. This is not a formulation shared with other countries in the world, including practically all of Europe. Considerations of risk have equally to do with societal risk. That includes the notion of the family, and what it means to have a ‘designer baby.’

“These were not diseased babies Dr. He was trying to cure. The motivation for the intervention was that they live in a country with a high stigma attached to HIV/AIDS, and the father had it and agreed to the intervention because he wanted to keep his children from contracting AIDS. AIDS shaming is a fact of life in China, and now it won’t be applied to these children. So, are we going to decide that it’s OK to edit as-yet-to-be children to cater to this particular idea of a society?

“It’s been said that ‘the genie is out of the bottle’ with germline human genome editing. I just don’t think that’s true. After all, we have succeeded in keeping ‘nuclear’ inside the bottle. Humanity doesn’t lack the will, intelligence, or creativity to come up with ways for using technology for good and not ill.

“We don’t require students to learn the moral dimensions of science and technology, and that has to change. I think we face similar challenges in robotics, artificial intelligence, and all kinds of frontier fields that have the potential to change not just individuals but the entirety of what it means to be a human being.

“Science has this huge advantage over most professional thought in that it has a universal language. Scientists can hop from lab to lab internationally in a way that lawyers cannot because laws are written in many languages and don’t translate easily. It takes a very long time for people to understand each other across these boundaries. A foundational concept for human dignity? It would not be the same thing between cultures.

“I would like to see a ‘global observatory’ that goes beyond gene editing and addresses emerging technologies more broadly.”

To learn more:

Technology and Public Purpose project, Belfer Center for Science and International Affairs, Harvard Kennedy School of Government, https://www.belfercenter.org/tapp/person

Concluding statement from the Second International Summit on Human Genome Editing. http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11282018b

A global observatory for gene editing: Sheila Jasanoff and J. Benjamin Hurlbut call for an international network of scholars and organizations to support a new kind of conversation. https://www.nature.com/articles/d41586-018-03270-w

Building Capacity for a Global Genome Editing Observatory: Institutional Design. http://europepmc.org/abstract/MED/29891181

Glenn Cohen’s blog: How Scott Gottlieb is Wrong on the Gene Edited Baby Debacle. http://blog.petrieflom.law.harvard.edu/2018/11/29/how-scott-gottlieb-is-wrong-on-the-gene-edited-baby-debacle/

Gene-Editing: Interpretation of Current Law and Legal Policy. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5651701/

Forum: Harvard T.H. Chan School of Public Health event on the promises and challenges of gene editing, May 2017: https://theforum.sph.harvard.edu/events/gene-editing/

Petrie-Flom Center Annual Conference: Consuming Genetics: Ethical and Legal Considerations of New Technologies: http://petrieflom.law.harvard.edu/events/details/2019-petrie-flom-center-annual-conference

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Study Abroad

Pros and Cons of Gene Therapy – Essay Tips

August 16, 2020 //  by  Amit Kumar

Genes are the most important part of the body of any individual as these are responsible for the appearance of a person and the behaviour. Genes are essentially nitrogenous codes that take part in the formation of chromosomes, the most important abstract.

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Over the year, genetic problems and gene-related illnesses are very common among the people and newborn babies. These disorders can cause the life of an individual to be limited.

Some of these problems show the symptoms pretty early, where there is a minimum chance of survival for the problematic one. Gene Therapy can be caused by an extra gene, one less gene, or a defective gene. Gene Therapy is one solution to this problem. 

Gene Therapy involves the removal of an extra gene, the addition of one gene in place of missing one, and replacement of the defective gene with the new one.

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Gene Therapy can give life to an individual and make them an average person. Long-Lasting and timeless effects of gene therapy are hoping to the person who is facing this problem. Improved quality of life is the result of Gene Therapy.

We’ll go through some pros and cons of Gene Therapy. 

Table of Contents

Gene Therapy – Pros

The favourable factors of Gene Therapy are:

It Is a Hope for Those Suffering from Genetic Problems:

Many newborn babies are genetically unwell because of which these kids can’t survive more as their immune system is not super enough to overcome this problem. Through gene therapy, we can make these death rates lessen and prevent parents from suffering from heartbreaking moments.

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Lessen or Removes the Abnormality:

The reduction and elimination in the gene therapy can save lives and can prevent the irregularity or discomfort grow. 80% of the diseases in this world are in the field of genes. Replacements of cells or chromosomes that are defective can relieve an individual. This medical treatment is magic.

Equal Applications for Other Groups of Living Things:

This medical remedy is not, particularly for human beings. This can also be implemented on animals as well as plants. This medical treatment affords the premise for the extension in lifespan by stabilizing their health and genetic situations occurring under these living things. This method can help us to grow more in the field of food. 

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Technological Therapy: 

In previous centuries, most of the cure was based on natural herbs. Now, in this century, gene therapy is one of those medical treatments that are purely based on technology, and it is the first technologically medical treatment to cure the defect which was once thought, totally impossible. 

Offers Large Scale Treatment with Enhanced Health and Increased Lifespan:

Heredity problems are prevalent nowadays. The person with a gene problem is probably inheriting his generation a disease from which he is suffering. Gene therapy plays a role in a big block from spreading this type of defect.

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This medical treatment enhances health by improving many of the body’s errors causing by genes problems. This therapy is the key to most of the health benefits if you’re suffering from anyone.

To make up long, the genetically unwell person should consult the doctor. By changing, replacing, and removing the abnormal genes can increase his/her lifespan.

Gene Therapy – Cons:

Where there are pros, there are cons. Every positive thing has something negative, which makes it complete. Gene therapy also goes through some unfavourable conditions. Genetically unwell man or woman inherits disease to their generation, which is not good at all.

Gene Therapy is very Expensive: 

Numerous gene therapy alternatives can be appropriately processed now, but each therapy begins at a very high price. For example, if you want your colour blindness to be treated by gene therapy, it will cost $1 million.

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Even the lowest price of gene therapy is $200,000. An average person can’t afford this much amount. People with a strong financial background can only enjoy this comfort.

Gene Therapy Can Be Unreliable:

When a new substance is introduced into the gene of a person, there are chances that the gene may not accept it and make it its antibody and attack it by reacting negatively.

The hassle with this feature is that the enzyme used to inspire the switch of genetic records may be removed with the aid of using the immune gadget earlier than it has the danger to workers. There can be problems with replication that restrict the effectiveness of the treatment.

Gene Therapy Requires Ongoing Treatment to Be Effective:

After gene therapy, you must have to take medicines recommended by your therapist. Many of the people who went through gene therapy had seen that their treatment was not showing further improvements as it stopped just because they’ve stopped taking medications.

Gene Therapy Can Have Uninsured Results:

Gene therapy can create more complicated and complex problems than the one which you were facing before this medical treatment. These are very microstructures, and treatment can be complicated for therapists, and anything can go wrong with the treatment and a person going through this treatment can face more problems.

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Gene Therapy Can Cause Unintended Complications and Side Effects:

No such notes are containing the information that gene therapy can also cause side effects if applied. As these are the microstructures and the building blocks of the normal behaviour of body and mind, the therapy can cause unintended complications and side effects which can affect the future generation of that person badly. It can lead to toxic diseases or make your life wear off an extended period.

Conclusion:

This technological Gene Therapy is a ray of light for the whole of mankind in this world. Where gene therapy is essential for an individual to survive who is suffering from any genetic problem, by the side, it also has some side effects which can ruin the whole life.

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If it is done correctly, it can increase your lifespan with a healthier body or vice versa. This article ties up under the topics of Gene Therapy with its pros and cons.

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• University of Wisconsin-Madison

An electrifying discovery may help doctors deliver more effective gene therapies

Departments:, focus areas:.

In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering at the University of Wisconsin-Madison has developed a stimulating method for making the human body more receptive to certain gene therapies.

The researchers exposed liver cells to short electric pulses—and those gentle zaps caused the liver cells to take in more than 40 times the amount of gene therapy material compared to cells that were not exposed to pulsed electric fields. The method could help reduce the dosage needed for these groundbreaking treatments, making them much safer and more affordable. The research appears in the April 30, 2024, issue of the journal PLOS One .

Gene therapy is one of the most promising technologies in medicine: By replacing, altering or introducing new genetic material into a patient’s cells, doctors may be able to cure or compensate for genetic diseases, including cystic fibrosis, sickle-cell disease, hemophilia, diabetes and many others. One of the bottlenecks in gene therapy, however, is delivery, or getting the right dose of genetic material into the target cells. The UW-Madison research suggests applying a moderate electric field which leaves no lasting damage, could help in creating more effective therapies.

The project began almost a decade ago with Hans Sollinger , a world-renowned transplant surgeon at UW-Madison. He had developed a gene therapy treatment for Type 1 diabetes, an autoimmune disease that attacks the pancreas, the organ that produces insulin.

In his treatment, the genetic code, or DNA, for insulin production is delivered into liver cells using an adreno-associated virus (AAV) that assists in transporting the therapeutic genes across the cellular membrane. This genetic code can then take up residence in liver cells, producing insulin without being attacked by the immune system in the pancreas.

While Sollinger had proof of concept that the therapy worked, he believed the future of the treatment hinged on delivery. He turned to Susan Hagness and John Booske , both UW-Madison professors of electrical and computer engineering , who have experience treating human cells with electrical pulses. “What we started talking about was local, targeted delivery and whether there was a way of getting the treatment DNA directly into the liver without passing it through the entire body and triggering the immune system,” says Hagness. “And whether we could use electric pulses in order to make this delivery process more efficient and dramatically reduce the dose needed.”

Research has shown that exposing cells to electric fields can often increase the ability of molecules to move through the cell membrane into the interior of a cell. So in the engineers’ study, ECE Ph.D. graduate student Yizhou Yao sought to determine whether the technique would increase the penetration of virus particles into liver cells. Using human hepatoma cells, a model system for studying the liver, Yao exposed batches of the cells to various concentrations of the AAV gene therapy virus particles, or vector, each containing a fluorescent green protein. She used a pair of electrodes to deliver an 80-millisecond electric pulse to each sample, then incubated the cells for 12 hours.

When she examined the results, 48 hours later, under a fluorescence microscope, Yao found that in the cells that had not received the electrical pulses, only a small percentage of them were glowing green, indicating they had been successfully penetrated, or transduced by the AAV particles. In stark contrast, those cells that had received a zap accumulated about 40 times the amount of the fluorescent green proteins delivered by the AAV.

Booske says the team has yet to discover exactly how the process works at the molecular level. “There’s enough known about electric pulsing that I think we could confidently state that it is opening nanopores through the cell membrane,” he says. “But then Yao got this remarkable result, and it dawned on us that virus particles are in general bigger and more complex than bare molecular particles and they already have their own way of getting inside cells. So we don’t really know if it’s the pores opening that has anything to do with it directly or indirectly.”

Sollinger passed away in May 2023 but the team says his legacy will live on through the ongoing research on this project and the work of other groups. The electrical engineering researchers are pursuing next steps with external funding and are optimistic that ultimately the technique will translate into clinical trials.

Yao, who will graduate in 2024, says she knew the study would be transdisciplinary, but didn’t realize just how far it would go. “I am an electrical engineer by training, and I don’t have a biology background,” she says. “Before this, the last time I used a microscope was in high school! It was quite a steep learning curve, learning to culture cells and carry out biology protocols. But I really enjoyed this project and liked its ultimate goal, which is to make the world a better place. That’s exactly what I want to achieve. And it has given me an advantage; while I still belong to ECE, I’m now half biologist!”

Susan Hagness is the Philip Dunham Reed Professor and chair of the Department of Electrical and Computer Engineering at the University of Wisconsin-Madison.

John Booske is the Keith and Jane Morgan Nosbusch Professor Emeritus in Electrical and Computer Engineering.

Other authors include Robert W. Holdcraft of the Cincinnati Children’s Hospital Medical Center.

Featured image caption: Electrical engineering PhD student Yizhou Yao had to develop a whole new set of skills to research the effects of electric pulses on the receptivity of liver cells to new types of gene therapy. Credit: Joel Hallberg.