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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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​Gene Therapy

Gene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patient’s cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.

Gene therapy. Gene therapy is a direct way to treat genetic conditions as well as other conditions. There are also other related approaches like gene editing. There are many different versions and approaches to gene therapy and gene editing. It all rests on understanding how genes work and how changes in genes can affect our health. Researchers all over the world are studying many different facets of gene therapy and gene editing.

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Guest Essay

The Future of Medicine Is Unfolding Before Us. Are We Nurturing It?

By Elizabeth Currid-Halkett

Dr. Currid-Halkett is a Guggenheim fellow and professor of public policy at the University of Southern California.

On Jan. 8, 2020, as I was parking my car, I got a long-awaited phone call from one of my son’s doctors. She informed me that our 7-month-old son, Eliot, had Duchenne muscular dystrophy, a fatal neuromuscular disease.

I can still remember the way the Los Angeles winter sunlight hit the dashboard. I can see my neighbor walking up her steps with groceries, a leaf falling, oblivious to the devastation below. “Life changes in an instant,” Joan Didion wrote. “The ordinary instant.” Our son had a fatal illness. He would die before us.

D.M.D. prevents the production of dystrophin, a protein needed to protect and repair muscle cells. It is caused by a genetic mutation on the X chromosome, thus the disease almost exclusively affects boys (one in 3,300). Over time, children with D.M.D. lose muscle mass and thus the ability to do basic things like run and walk. Eventually they lose their ability to breathe, and they experience heart failure. There is no known cure. While existing treatments have helped extend the life span of sufferers, they mainly focus on managing symptoms.

In my search for answers for how to save my son, I contacted Dr. Jerry Mendell, a now-retired neurologist at Nationwide Children’s Hospital in Columbus, Ohio, who was running clinical trials for an experimental gene therapy he developed to enable dystrophin production in boys with D.M.D. The treatment, now known as Elevidys, offered the prospect of not merely managing symptoms, but slowing the disease’s progression or even stopping it in its tracks — and potentially, for the first time in the history of this terrible disease, allowing boys with D.M.D. a chance to thrive.

Since I had that first conversation with Dr. Mendell (also a senior adviser for Sarepta, the maker of Elevidys), clinical trials for the gene therapy have had their ups and downs , and some adverse effects have been reported. But in June 2023, based on a two-part clinical trial, the Food and Drug Administration granted accelerated approval for the treatment for 4- and 5-year-olds who do not have other disqualifying conditions. The F.D.A.’s approval was contingent on continuing trials showing evidence of improved motor function, which had not yet been established.

Before Eliot received his treatment, he had difficulty going up stairs. He complained about being tired after walking only a block or two, even on Halloween, when candy ought to have motivated him. Hopping on one foot, a milestone for a 4-year-old, was impossible.

On Aug. 29, he finally received the one-time infusion. Three weeks later, he was marching upstairs and able to jump over and over. After four weeks, he could hop on one foot. Six weeks after treatment, Eliot’s neurologist decided to re-administer the North Star Ambulatory Assessment , used to test boys with D.M.D. on skills like balance, jumping and getting up from the floor unassisted. In June, Eliot’s score was 22 out of 34. In the second week of October, it was a perfect 34 — that of a typically developing , healthy 4-year-old boy. Head in my hands, I wept with joy. This was science at its very best, close to a miracle.

But the goal to offer this possible future to more patients with D.M.D. is in jeopardy. Sarepta is seeking F.D.A. approval to treat boys over 5 . Disagreements over the latest clinical trial’s results threaten to derail that outcome.

Moreover, what the F.D.A. decides to do next with Elevidys could set the tone for how it handles other emerging gene therapies for rare diseases. We can already see roadblocks that prevent more families from gaining access to these new treatments — from high costs and insurance challenges to dissent over how flexible regulators should be in interpreting clinical trial results and taking qualitative improvements into account. What is at stake with the debate around Elevidys is more than just the chance to give other boys with D.M.D. a more normal life. The challenges that we are witnessing with Elevidys are a harbinger of the fights we may see with gene therapies developed for other rare diseases.

There’s an opportunity to reduce those barriers now, while these treatments are still in their early phases. Every child afflicted with a life-threatening disease deserves the chance Eliot has been given.

The biggest obstacle to getting these treatments is cost. Gene therapies cost, on average, $1 million to $2 million . At $3.2 million per patient, Elevidys is the second-most-expensive drug in the world . Insurance companies would probably prefer not to foot the bill, and without full F.D.A. approval, insurance companies can refuse to cover these treatments by claiming they are medically unnecessary or experimental . Before Eliot’s treatment began, my insurance company initially said it would cover the cost but then started stalling on coverage and questioning the urgency of Eliot’s treatment. I was able to call Dana Goldman, the dean of the Sol Price School of Public Policy at the University of Southern California, where I work, to help me navigate the process. I was in the rare position to marshal resources and assistance to pressure my insurance company into covering Elevidys. Across the country, physicians are fighting denials and seeking appeals for their young patients.

Dr. Goldman has argued that one way to incentivize insurance companies to cover the high costs of treatments like gene therapies is to amortize how much the companies pay over time if the effectiveness of such treatments does not last (analogous to a pay-for-performance model). Another option is for pharmaceutical companies to offer a warranty that gives a prorated refund to the insurance company if a patient needs to return to prophylaxis treatment within a certain number of years. Costs are an especially frustrating problem for rare diseases like D.M.D., for which the extremely small patient population deters companies from investing money and resources to develop new treatments. Some experts believe the federal government ought to do more to directly complement research funding for rare diseases , as it has through the Orphan Drug Act for over four decades. The government could also defray the cost to consumers by offering subsidies directly to patients.

There’s another big role the government can play to accelerate gene therapies besides intervening in costs, and that’s to make the wheels of regulatory approval for these drugs less onerous. Flexibility doesn’t have to come at the cost of safety. The F.D.A. acted swiftly to approve an antiretroviral drug for H.I.V. in the 1980s and the Covid vaccines in December 2020, saving millions of lives without putting people in harm’s way.

But Elevidys is a case study in how the F.D.A. can get in its own way. D.M.D. patients 4 or 5 years old received access to the drug under fast-tracked approval, the first time a drug was approved under this new framework. But this was reportedly only because Peter Marks , the director of the F.D.A.’s Center for Biologics Evaluation and Research, disagreed with his own staff’s rejection . Current concern over Elevidys’s approval for boys over 5 focuses on the most recent clinical trial results , which showed older boys, whose muscular decline is further along, did not improve on motor function as measured by the North Star Ambulatory Assessment after treatment. However, as Sarepta has noted, they still saw gains in their ability to rise from the floor and walk 10 meters, indicating possible slowing of the disease that could significantly improve and extend their lives.

Detractors suggest this improvement is not enough to meet the bar for approval. This is a common problem for rare disease trials because they often consist of very few participants. In such cases, a narrow focus on numbers ignores the real quality-of-life benefits doctors, patients and their families see from these treatments. During the advisory committee meeting for Elevidys in May 2023, I listened to F.D.A. analysts express skepticism about the drug after they watched videos of boys treated with Elevidys swimming and riding bikes. These experts — given the highest responsibility to evaluate treatments on behalf of others’ lives — seemed unable to see the forest for the trees as they focused on statistics versus real-life examples.

The F.D.A. can have a more flexible view of treatment efficacy without losing focus on safety. As with any drug, whether for migraines or asthma, there will be a spectrum of effectiveness. The same will be true of all gene therapies, and the F.D.A. should reconsider the metrics it uses to green-light these treatments now, before it potentially leaves thousands of patients in the lurch, out of access to something lifesaving.

Gene therapy is the future of medicine. Our bureaucracy and insurance companies should not hinder patients from receiving pioneering treatments that could transform their lives. As parents, we are not asking for the moon. We just want our children to live.

Elizabeth Currid-Halkett is a Guggenheim fellow and professor of public policy at the University of Southern California.

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Welcome to Gene Therapy

A key title for the exploration of new genetic and stem cell therapy techniques

Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery

• Sarah Klinnert
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Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo

• Gabriela Toro Cabrera
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• Christian Mueller

Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases

• Duncan J. Stewart
• Albert Gianchetti
• Rickey R. Reinhardt

A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies

• Shadi Saleh
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Implications of maternal-fetal health on perinatal stem cell banking.

• Mehri Barabadi
• Rebecca Lim

Acoustically targeted noninvasive gene therapy in large brain volumes

• Shirin Nouraein
• Sangsin Lee
• Jerzy O. Szablowski

Long-term effects of a fat-directed FGF21 gene therapy in aged female mice

• Jacqueline M. Anderson
• W. David Arnold

p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma

• Naomi Muramatsu
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Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease

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A multinational survey of potential participant perspectives on ocular gene therapy

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Precision ophthalmology: a call for Africa not to be left in the dark

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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation

• Maria Elisabeth Kalland
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A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice

• Felix Krause
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Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate

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Analytical characterization of full, intermediate, and empty AAV capsids

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Gene Therapy: Risks and Benefits Research Paper

Introduction, summary of the mechanisms of gene therapy, discussion of paper and critique of the methods used, argument and future experiments proposed, works cited.

Gene therapy is the “insertion or removal of genes which can also be alternated within the cell or tissues of an organism for purposes of treating diseases” (Cross & Burmester). All over the world, “the technique is best known for the correction of defective genes so as to treat diseases; the most common procedural form of gene therapy involves the insertion of the functional gene in order to replace the mutated gene within an organism” (Cross & Burmester).

Over the recent past use of gene therapy has revolutionized the treatment approaches, and research on this subject is justified because of the great potential that this technique offers. Similarly, further research will also shed light on possible risk factors that are associated with this method of treatment. The purpose of this paper is to undertake a cost-benefit analysis of gene therapy as a treatment option.

Germ-line gene therapy

Under normal circumstances, germ-line gene therapy procedures involve alternating and replacing all defective genes within the body of an organism (Walesgenepark.co.uk). In this case, functional genes are isolated and inserted into the interior lobes of the reproductive tissues or cells of the relevant organism. Considerably, this therapy operates on the basis that fresh and healthy genes get inoculated into the gametes of an organism in order to reduce the risks of later transfer of the defective genes to the next generation of the organism (Walesgenepark.co.uk).

Moreover, there is the involvement of complete alteration of the genetic makeup of early-stage blastomere so as to enhance changes of the genetic codes which can be passed on from generation to generation. Secondly, germ-line therapy also involves alteration of the gametes before fertilization since, after fertilization, the characteristics are passed on to the offspring’s (Walesgenepark.co.uk).

Somatic gene therapy

Generally, “somatic gene therapy involves the process of alteration of the genetic makeup of somatic cells (i.e., all body cells except sex cells) of an individual” (Rothchild, Laura & Lauren). Contrary to germ-line therapy, there is no transfer of these cellular changes to the next generation of the organism, simply because they are neither sex cells nor gametes which fuse during fertilization (Rothchild, Laura & Lauren). The main focus on the process involves changing the arrangements of the genetic codes of the specific cells using either an in-vitro or ex-vivo DNA delivery system. This technique in today’s life can be employed medically in treatments of a variety of diseases, including; hemophilia, muscular dystrophy, among many others; currently, it is used for cancer treatments (Rothchild et al.).

Telomerase inhibition strategies

In humans, it’s evident that the telomerase RNA abbreviated (hTR) plays an important role in anticancer therapy. This hTR can be used as an anticancer either individually or in combination with another human telomerase reverse transcriptase (hTERT) (Li, Li, Yao, Geng, Xie, Feng, Zhang, Kong, Xue, Cheng, Zhou & Xiao 4). Current findings indicate that when these two agents combine with the recombinant adenovirus, another nucleotide called small-interfering RNA (siRNA) is formed (Li et al. 4).

Furthermore, it has been established that the levels of telomerase activities together with mRNA, hTR are greatly reduced by the activities of the recombinant adenovirus resulting in inhibition of Xenograft tumor growth (Li et al. 4). This implies that siRNA, which is specifically expressed in recombinant adenoviruses, is the best tool to be used as an anticancer and also in the treatment of oral squamous cell carcinoma (OSCC) (Li et al. 4). The major advantage of this technique is that the anticancer effect on OSCC is virtually accomplished by cellular proliferation in addition to cellular apoptosis (inhibition of tumor angiogenesis) (Li et al. 4).

Monoclonal antibodies in target therapies of breast cancer

Globally, breast cancer is known to be a killer disease that largely affects females; the major causative agent in about 10% of world breast cancer cases is the mutation of the gene, which is inherited from any of the parents (Grammatikakis, Zervoudis & Kassanos 640). Furthermore, the most effective known therapeutic alternative diagnosis or treatment of breast cancer globally is the use of gene therapy. Some of the procedures used in treatment include molecular chemotherapy, antiangiogenic gene therapy, among many other therapies currently being used (Grammatikakis et al. 640).

bacteria-mediated anti-angiogenesis therapy

Most of the recent studies have revealed that there are some bacterial species that are capable of colonizing solid tumors. This inherited characteristic, however, can further be enhanced via genetic engineering, developing a natural anti-tumor activity that always enables the specific bacteria to transfer its therapeutic molecules directly into the target cells (Gardlik, Behuliak, Palffy, Celec & Li 7). There are few completed studies that have completely documented the anti-angiogenesis process, which is basically a bacterial mediated therapy for cancer (Gardlik et al. 7).

There are four recognized approaches that scientists use when utilizing the use of bacteria in cancer treatments; these include anticancer therapeutic-autofiction, DNA vaccination, transkingdom RNA interference, and alternative gene therapy (Gardlik et al. 7). Notable to mention is that the major primary goal of all these approaches is that they all focus on stimulation of angiogenesis suppression.

Based on the evidence of this paper, the discussion focuses on the argument of whether gene therapy is effective when the cost-benefit analysis is undertaken. Personally, I totally agree with the essay topic that gene therapy has more benefits than costs since it has been successful in the treatment of most chronic cancerous diseases. Gene therapy usually works by relying on immunotherapy and the use of vector organisms like viral particles to modify the genome of the host cell that triggers an immune response, which finally destroys the cancer cells present in the body (Cross & Burmester).

By using gene therapy, many cancerous diseases, i.e., lung, prostate, pancreatic cancers, have a higher chance of being completely treated. As such, gene therapy is now emerging to be the most common preferred treatment of choice because of its efficacy over other treatment methods (Cross & Burmester). The gene treatment also allows the use of a single vector or a combination of several vectors aimed at achieving optimal results (Cross & Burmester).

Currently, further research on gene therapy is still ongoing, and various clinical trials have so far been completed, which now has led to the evolution of vaccine productions (Cross & Burmester). The vaccines are promising to be the most effective technique since they only require autologous cells to have them manufactured, although this has many cost implications; this is one of the disadvantages of this technique. The second disadvantage is that very few hospitals globally are capable of having such vaccines manufactured because of the costly technology associated with the production of the vaccine. All these factors ultimately limit the availability of the vaccine as a viable treatment option.

A recent research study provides findings that show evidence of secondary gliomas stem cell, which originates from an astrocytic tumor that contains a genetic mutation that possesses the tumor suppressor gene recognized as p53 (Cross & Burmester). As part of the procedure, it was proposed in the experiments that in order to induce apoptosis of tumor cells, one must incorporate the use of an integrated suicide factor together with the adenovirus-mediated transfer of p53 (Cross & Burmester). Considerably, the main strength of this approach is supported by the fact that p53 mediate apoptosis follows two distinct and separate pathways reducing pathogenicity (Cross & Burmester).

Gene therapy technology has brought a great 21st-century revolution to the modern system of disease treatments, precisely when it comes to cancer treatments. It is remarkable to note that the development of anticancer treatments by modified immunotherapy and gene therapy, among others, has helped to cure many cases of cancers and saved many others from death. However, through gene therapy, many victims of cancer have attained a prolonged lifespan after being subjected to this treatment.

Scientifically, the principle behind gene therapy when it comes to cancer treatment is that a successful cancer treatment involving therapeutic modality always aims at real activation of death pathways within the cancer cells.

Therefore, there is no doubt that for cancerous diseases, the development of genetic engineering and specific cancer vaccines are proving to be the most effective treatment approaches. There are hope and confidence that in the near future, all obstacles encountered during the first generation cancerous and precancerous treatments will be eliminated by the development of second-generation modern therapeutic intervention as far as disease (cancer) treatments is concerned (Cross & Burmester).

Cross, Deanna., & James, Burmester. “ Gene Therapy for Cancer Treatment: Past, Present and Future .” Clinical Medicine and Research . 2006. Web.

Gardlik, R., Behuliak, M., Palffy, R., Celec, P., & Li, C. “Gene therapy for cancer: bacteria- mediated anti-angiogenesis therapy.” Clinigene Current Gene Therapy Weekly (2011): 7.

Grammatikakis, I., Zervoudis, S., & Kassanos, D. “Synopsis of new antiangiogenetic factors, mutation compensation agents, and monoclonal antibodies in target therapies of breast cancer.” Clinigene Current Gene Therapy Weekly (2011): 7.

Li, Y., Li, M., Yao, G., Geng, N., Xie, Y., Feng, Y., Zhang, P., et al. “Telomerase inhibition strategies by siRNAs against either hTR or hTERT in oral squamous cell carcinoma.” Clinigene Current Gene Therapy Weekly (2011): 4.

Rothchild, Allisa., Laura, Martin., & Lauren, Lubrano. “Gene Therapy and the Gametes.” Somatic Gene Therapy . 2010. Web.

Walesgenepark.co.uk . “What is Germline Gene Therapy.” Wales Gene Park . 2011. Web.

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IvyPanda. (2024, February 17). Gene Therapy: Risks and Benefits. https://ivypanda.com/essays/gene-therapy-risks-and-benefits/

"Gene Therapy: Risks and Benefits." IvyPanda , 17 Feb. 2024, ivypanda.com/essays/gene-therapy-risks-and-benefits/.

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IvyPanda . 2024. "Gene Therapy: Risks and Benefits." February 17, 2024. https://ivypanda.com/essays/gene-therapy-risks-and-benefits/.

1. IvyPanda . "Gene Therapy: Risks and Benefits." February 17, 2024. https://ivypanda.com/essays/gene-therapy-risks-and-benefits/.

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IvyPanda . "Gene Therapy: Risks and Benefits." February 17, 2024. https://ivypanda.com/essays/gene-therapy-risks-and-benefits/.

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Gene Therapy: Concept and Types

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Published: Feb 12, 2019

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• v.29(8); 2021 Aug 4

Updating the ethical guidance for gene and cell therapy research participation

Stephanie solomon cargill.

1 Center for Health Care Ethics, Saint Louis University, St. Louis, MO, USA

4 Chair of Castle IRB, Wildwood, MO, USA

Andrea Eidsvik

2 Center for Health Care Ethics, Saint Louis University, St. Louis, MO, USA

Marilyn Lamm

3 Institutional Biosafety Committee (IBC), Clinical Biosafety Services, Wildwood, MO, USA

Since the US Food and Drug Administration (FDA) approved the first gene therapy in 2017, five additional modalities of gene and cell therapy (GCT) have been approved 1 and 3,032 clinial trials worldwide employ GCT technologies targeting a broad array of diseases and conditions ( https://asgct.careboxhealth.com ). Many of these studies target diseases or conditions that have currently approved clinical treatments and are also being offered to patients before they have tried and failed these alternative treatment options, thus potentially conflicting with existing ethical standards. We argue here that the current trend toward enrolling patients into early-phase GCT trials as a front-line approach is not unethical, but nevertheless problematic, unless it is approached with caution and consistency. Guidance is needed to assist researchers, sponsors, and review bodies in determining whether to offer GCT trial participation as a front-line option to patients and the key information to disclose to them if or when they do.

Historically, enrollment of participants in early-phase GCT trials proceeded with extreme caution. 2 , 3 , 4 Conditions for ethical enrollment required that participants have a very serious unmet medical need and only when patients have no other options (i.e., the “only line” approach) or when patients are treatment-refractory (i.e., the “last line” approach). Moreover, it has been considered unethical to offer participation to treatment-naive patients or patients who are on other treatments but who might desire to enroll in GCT trials instead (i.e., a front-line approach).

The absence of “viable and reasonable” alternatives is a part of nearly all historic and current ethical criteria for early-phase GCT clinical trial participation. While this threshold is clear for diseases with no treatment options, it becomes more complex when applied to diseases or disorders that do have clinical alternatives. In these cases, the ethical standard is frequently assumed to require attempting and failing any clinically approved treatment options available. While equating any approved standard of care with a viable and reasonable alternative is understandable both for ease of application and intuitive appeal, this equation is problematic for several reasons.

First, this interpretation is not in line with existing guidance on the issue, including from the National Academies of Science, 5 the FDA, 6 and bioethicists, 7 , 8 who emphasize that the standard is meant to be based on judgments about alternatives in particular circumstances, not applied to any alternatives. While standard of care treatments are inherently more predictable than experimental ones because of a history of evidence and clinical usage, they should not thereby be assumed to be less risky or more beneficial than clinical trial alternatives. Many approved interventions, especially in the treatment of rare diseases, cancer, and chronic diseases, are suboptimal in many ways and carry with them significant risks as well as limited benefits. Patient lifestyle, stage of disease progression, and disease manifestation, as well as socioeconomic and psychological circumstances, also impact the success rate and acceptability of various existing treatments.

Further, in the decades since the tragic deaths of Jesse Gelsinger and Jolee Mohr, the landscape of GCT has changed significantly. More is known about GCT and it “is increasingly becoming an important tool in the medical armamentarium for medical treatment.” 9 While GCT technology is still relatively new and evolving, it is a mistake to consider all GCT clinical trials as characterized by the global uncertainties of decades ago. Vector designs and packaging systems have evolved to minimize risks of insertional mutagenesis, immune responses, cytotoxicity, and recombination with wild-type viruses, leading to improved safety. This increase in knowledge provides a good reason to revisit the standard ethical approach as it undermines the assumption of global uncertainty about the risks and benefits of GCT clinical trials in comparison to existing treatments. In 1997, bioethicist Leroy Walters consequently proposed that, as the field of gene therapy matured, the requirement that there be no effective alternative therapy may need to be relaxed. 7

While there are good reasons to challenge the standard approach, we must be wary of abandoning it completely. If we uncritically enroll participants in novel GCT trials as a front-line approach, we risk overromanticizing the anticipated (but as yet unproven) benefits and underplaying their risks and uncertainties. Without close consideration of the available standard alternatives in each case, optimism among researchers and patients about GCT is likely to overshadow the standard care even in cases when the approved alternatives may be the more beneficial treatment options for patients. The potential and expectation for GCT has driven and pressured researchers to advance the field for new clinical treatments as quickly as possible. We agree with critics who urge that optimism should be balanced with caution. 7 Thus, an evaluation will need to be made that takes into consideration the specific factors of a GCT study to determine whether front-line enrollment is ethical. For this more nuanced judgement, a framework of considerations is needed to guide enrollment decisions, ensuring that the choices made are consistent and justified.

We suggest four considerations that must be applied by researchers and review bodies to determine when enrollment in GCT trials can ethically be offered as a front-line approach to participants.

1. Evidence for safety and efficacy of GCT

While first-in-human phase I GCT studies that only have previous evidence of safety and efficacy from animal models should remain on the precautionary end of the spectrum, often early phase trials are assessing well-studied components with only slight modifications that classify them as experimental. While any novelty introduced into a clinical trial design has the possibility to alter its safety or efficacy profile, studies that use well-studied investigational products should require a lower threshold for comparison to existing treatments as those that involve more novel components. Further, dose escalation studies, where it is likely that participants may receive a subclinical dose, should be considered more cautiously than dose expansion trials where the range between the minimum effective dose and the maximum tolerated dose has been established and some efficacy can be expected for all participants.

2. Evidence for safety and efficacy of clinical alternatives

The safety and efficacy of existing clinical alternatives should be compared with the safety/efficacy profile produced from consideration number one. This should include considerations of the side effects, toxicities, clinical effectiveness, length of effectiveness, etc. of the standard alternatives. Comparisons with interventions approved through alternative mechanisms (such as accelerated approvals by the FDA 6 ) should acknowledge that the safety and efficacy thresholds required for approval may have been lower than in standard approval mechanisms.

3. Impact of therapeutic windows or interactions on timing of research and clinical alternatives

Many GCT trials have therapeutic windows for eligibility. Patients with degenerative diseases can only participate in these trials prior to a certain point of disease progression or a certain age. 10 When there are clinical alternatives, an important consideration is whether these alternatives can be delayed, paused, or continued and with what drawbacks, in light of the potential benefits, of participating in a trial within its therapeutic window.

4. Value-based and practical patient considerations concerning clinical alternatives

The risk-benefit comparison between GCT trials and the clinically approved alternatives involves not only information regarding safety and efficacy, but also subjective or context-based considerations of interest to patients. Echoing Kimmelman, 8 we suggest involving patient and disease advocacy organizations to provide input about the contextual risks and burdens of clinical alternatives. 8 It is crucial for researchers and isponsors to be aware of these concerns and refer to them explicitly in their justification for offering front-line GCT, especially when consideration number two may indicate that standard of care is preferable. We recognize that these stakeholders may embrace the romanticization of GCT that could lead to studies that pose an unacceptable risk to participants. On the other hand, with well-supported guidance from disease groups and stakeholders, both the permission to enroll in these studies as well as strategies to explain the alternatives transparently (see below) can prove these enrollment decisions are ethically justified based on the autonomy of the participants.

Ultimately the ethical balance for GCT enrollment is struck in two ways. First, the historical ethical standard of excluding front-line participants should remain only as a default, with the burden of justification placed on enrolling front-line participants. If the four considerations above demonstrate that the clinical alternatives are not clearly superior to the GCT approach under study, then early-phase GCT trials can be justified as a front-line option. Second, when GCT research is being offered as a front-line approach, the ethical onus shifts from the enrollment to the consent process. To ethically enroll patients into early GCT studies who have other clinical alternatives, these alternatives must be laid out clearly, honestly, and specifically in the consent process. This responsibility lies with both those who design and deliver the consent process, as well as those who review it. In this way, caution is preserved while acknowledging the evolving state of the field of GCT.