Category Archives: Gene therapy

Hundreds of clinical trials are underway studying the technologys potential use in a wide range of genetic disorders, cancer and HIV/AIDS. There is some debate over whether or not the US already has approved its first gene therapy treatment.

In August 2017, the Food and Drug Administration (FDA) approved a cancer therapya CAR-T treatment marketed as Kymriahthat uses a patients own T cells and is a variation of the gene therapy that is being developed to treat single-gene diseases. The T cells are extracted and genetically altered so that they have a new gene that codes for a protein, known as a chimeric antigen receptor (CAR), that is a hybrid of two immune system proteins. One part guides the cells to the cancer cell targets and the other alerts the immune system. The cells, programmed to target and kill leukemia cells, are then injected back into the patient. Another CAR-T treatment, marketed as Yescarta, was approved for adults with aggressive forms of non-Hodgkins lymphoma in October 2017.

Some in the scientific community have pushed back against the idea of calling Kymriah or Yescarta true gene therapies, since they dont actually repair or replace a deficient gene. Instead, they say the most likely candidate to gain the first US approval is Luxturna, a one-time treatment that targets a rare, inherited form of blindness. A key committee of independent experts voted unanimously in October 2017 to recommend approval by the FDA for the treatment developed by Spark Therapeutics. The FDA is not bound by the panels decision, though the agency traditionally acts on its recommendations.

Hundreds of research studies (clinical trials) are underway to test gene therapies as treatments for genetic conditions, cancer and HIV/AIDS. ClinicalTrials.gov, a service of the National Institutes of Health, provides easy access to information about clinical trials. There is also a list of gene therapy clinical trials that are accepting (or will accept) participants. Among the studies and research:

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Gene Therapy Archives | Genetic Literacy Project

Gene therapy is a technique throughwhich genes are added or replaced to treat or prevent disease.

Our genes, which hold the code for all of our body's functions, aremade of DNA. Damage to DNA, such as a mutation, is an underlying cause of thegenetic defects that lead to cancers, blood disorders, and other conditions.Gene therapy delivers DNA into a patients cells to replace faulty or missinggenes or add new genes in an attempt to cure cancer or make changes so thebody is better able to fight off disease.

Scientists are investigating a number of different ways to do this:

How does gene therapy deliver new genes into cells?

With gene therapy, the DNA for the new or corrected gene or genes iscarried into a patients cells by a delivery vehicle called a vector, typicallya specially engineered virus. The vector then inserts the gene(s) into thecells' DNA.

For patients, the process for delivering genes to cells is fairlysimple.

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What is Gene Therapy? - Dana-Farber/Boston Children's ...

The time is right for gene therapy.

Over the last decade, adeno-associated virus (AAV) has emerged as a highly promising and attractive approach to gene therapy. AAV is a common, naturally occurring virus that has been shown to be a well-toleratedand effective gene therapy delivery vehicle in clinical trials. Advances in AAV vector design and related dosing techniques that enable widespread gene delivery in the brain and spinal cord have made AAV particularly well-suited for the treatment of neurological diseases. Since the targeted cells in the central nervous system (CNS) are long-lived, non-dividing neurons, treatments delivered in a single dose could generate long-lasting, or even lifelong, benefits. More than eight years of durable expression has been seen in the human brain following treatment with an AAV vector.

Importantly, improvements in related technology and approaches have made AAV production more easily scalable and efficient to meet clinical and commercial requirements. Voyager diligently selects and optimizes AAV vectors that are best suited for each program. We continue to invest to advance the science and technology around the three key elements of AAV vectors: capsid, promoter and transgene. We also systematically develop and optimize delivery techniques that are best suited for a particular disease.

Members of our team have co-discovered many of the known naturally occurring AAV capsids, which are the outer viral protein shells that enclose the target gene or micro RNA cassette, and have also created promising genetically engineered AAV capsids. We have efforts underway to genetically engineer capsids to yield vectors with desirable properties, such as enhanced tissue specificity and improved delivery of genes to the brain and spinal cord.Efforts are also underway at Voyager to optimize novel AAV capsids that demonstrate enhanced blood-brain barrier penetration for the potential treatment of CNS diseases following systemic administration of the AAV gene therapy vector.

We then design the vector genome, or payload, that we intend to deliver as a therapeutic, as in the case of our Friedreichs ataxia program, or silence or knockdown, as in the case of our ALS and Huntingtons disease programs.

Identifying the optimal route of administration and delivery parameters, such as infusion volume, flow rate, vector concentration and dose and formulation for a specific disease are critical to achieving safe and effective levels of gene expression in the targeted region of the CNS. For Voyagers current pipeline programs, we are pursuing a surgical approach for direct injection into a targeted region of the brain, coupled with real-time MRI in the case of our advanced Parkinsons disease and Huntingtons disease programs, or injection into the cerebrospinal fluid for broader delivery to the cells within and surrounding the spinal cord for our ALS and Friedreichs ataxia programs.

Led by pioneers in AAV gene therapy and neuroscience, we are deeply committed to developing gene therapies for severe neurological diseases that have the potential to positively impact the lives of people living with these diseases. For more information about how we engage with patients and the advocacy community, please visit our patients and caregivers page.

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Gene Therapy | Voyager Therapeutics

All types of cancer cells appear to have at least one essential thing in common: They have faulty genes. At the center of every cell in our bodies, there is a nucleus containing thousands of genes made of DNA. Genes are coded instructions for making proteins, the molecules that control how cells work.

A cell with healthy DNA will perform its function in the body, create new cells as needed and destroy itself when it is damaged beyond repair. However, when a carcinogen such as asbestos damages the DNA in a cell, it may cause the cell to grow and divide out of control, leading to cancer.

Many researchers believe that just as faulty genes are the key to cancer formation, modified genes may be the key to cancer treatment. Mesothelioma researchers are hopeful that gene therapy will bring us closer to a cure for mesothelioma.

Gene therapy is a broad category that refers to several emerging treatment approaches involving the novel science of genetic modification. It wasnt until recently in 2017 that the U.S. Food and Drug Administration (FDA) approved a gene-therapy-based cancer treatment for the first time.

So far, most gene therapies tested for mesothelioma have shown either limited effectiveness or severe side effects and risks of complications. For this reason, all types of gene therapy for mesothelioma are experimental and only available through clinical trials.

The most obvious gene therapy approach is to fix the genetic fault that causes cells to become cancerous in the first place. To perform this medical feat, however, scientists have to overcome two major challenges.

First, researchers have not been able to pinpoint a specific gene that can stop the progression of mesothelioma in most patients. The likeliest candidates are natural tumor-suppressing genes that prevent genetic mutations or ensure mutant cells self-destruct before they grow into tumors. The p53 gene, the BAP1 gene and microRNA gene 16 have all been studied as genes that may be able to stop the progression of mesothelioma.

Second, inserting these tumor-suppressing genes requires a microscopic delivery vehicle, or vector, that can penetrate deep into a tumor. Genetically modified viruses and specially designed nanoparticles are both in development as gene therapy vectors.

Get help connecting with the nation's top mesothelioma doctors and cancer centers.

The same vectors that could carry tumor-suppressing genes could also insert artificial suicide genes into cancer cells.

If researchers can develop a vector that infects all the cells in a tumor while leaving the rest of the bodys cells alone, it would enable a special form of targeted chemotherapy called suicide gene therapy. The artificial suicide gene causes cancer cells to produce an enzyme that converts an otherwise harmless drug into a lethal toxin, so the drug kills cancer cells while leaving healthy cells unharmed.

Rather than trying to alter cellular DNA, some researchers instead focus on modifying deadly viruses to only kill cancer cells. This approach, known as virotherapy, was discovered by accident when doctors noticed many cancer patients who contract measles experience tumor regressions. Since then, scientists have been developing modified versions of the measles virus as an experimental treatment for several types of cancer, including mesothelioma.

In a 2016 trial of virotherapy for pleural mesothelioma patients, researchers were able to safely inject a special strain of the measles vaccine directly into the cancer site, potentially fighting the cancer through viral infection as well as provoking a natural immune system response against the cancer.

The most exciting recent development lies at the intersection of gene therapy and immunotherapy, another cutting-edge cancer treatment science. The first gene therapy for cancer approved by the FDA is known by the brand name Kymriah and generically referred to as CAR T-cell therapy. Kymriahs makers call it a living drug, because it is produced by extracting the patients own immune cells and reprogramming them to target cancer.

CAR T-cell therapy represents one of the first truly individualized and targeted cancer treatments, but it also has significant limitations: Kymriah is FDA-approved only for leukemia, it is extremely expensive, and it comes with the risk of severe side effects. Nevertheless, this technology has the potential to improve outcomes for mesothelioma patients in the future.

Last Modified September 25, 2018

Registered Nurse and Patient Advocate

Karen Selby joined Asbestos.com in 2009. She is a registered nurse with a background in oncology and thoracic surgery and was the director of a tissue bank before becoming a Patient Advocate at The Mesothelioma Center. Karen has assisted surgeons with thoracic surgeries such as lung resections, lung transplants, pneumonectomies, pleurectomies and wedge resections. She is also a member of the Academy of Oncology Nurse & Patient Navigators.

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Gene-Therapy - Experimental Mesothelioma Treatment

Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because thats what viruses evolved to do with their own genetic material.

The treatment, which was first tested in humans in 1990, can be performed inside or outside of the body. When its done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells. This is useful when only certain populations of cells need to be fixed. For example, researchers are using it to try to treat Parkinson's disease, because only part of the brain must be targeted. This approach is also being used to treat eye diseases and hemophilia, an inherited disease that leads to a high risk for excess bleeding, even from minor cuts.

Early in-the-body gene therapies used a virus called adenovirusthe virus behind the common coldbut the agent can cause an immune response from the body, putting a patient at risk of further illness. Today, researchers use a virus called adeno-associated virus, which is not known to cause any disease in humans. In nature, this agent needs to hitch a ride with an adenovirus, because it lacks the genes required to spread itself on its own. To produce an adeno-associated virus that can carry a therapeutic gene and live on its own, researchers add innocuous DNA from adenovirus during preparation.

In-the-body gene therapies often take advantage of the natural tendency of viruses to infect certain organs. Adeno-associated virus, for example, goes straight for the liver when it is injected into the bloodstream. Because blood-clotting factors can be added to the blood in the liver, this virus is used in gene therapies to treat hemophilia.

In out-of-the-body gene therapy, researchers take blood or bone marrow from a patient and separate out immature cells. They then add a gene to those cells and inject them into the bloodstream of the patient; the cells travel to the bone marrow, mature and multiply rapidly, eventually replacing all of the defective cells. Doctors are working on the ability to do out-of-the-body gene therapy to replace all of a patient's bone marrow or the entire blood system, as would be useful in sickle-cell anemiain which red blood cells are shaped like crescents, causing them to block the flow of blood.

Out-of-the-body gene therapy has already been used to treat severe combined immunodeficiencyalso referred to as SCID or boy-in-the-bubble syndromewhere patients are unable to fight infection and die in childhood. In this type of gene therapy, scientists use retroviruses, of which HIV is an example. These agents are extremely good at inserting their genes into the DNA of host cells. More than 30 patients have been treated for SCID, and more than 90 percent of those children have been cured of their disorderan improvement over the 50 percent chance of recovery offered by bone marrow transplants.

A risk involved with retroviruses is that they may stitch their gene anywhere into DNA, disrupting other genes and causing leukemia. Unfortunately, five of the 30 children treated for SCID have experienced this complication; four of those five, however, have beaten the cancer. Researchers are now designing delivery systems that will carry a much lower risk of causing this condition.

Although there are currently no gene therapy products on the market in the U.S., recent studies in both Parkinson's disease and Leber congenital amaurosis, a rare form of blindness, have returned very promising results. If these results are borne out, there could be literally hundreds of diseases treated with this approach.

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How does gene therapy work? - Scientific American

In this impressive, meticulously researched study of the exciting new developments in gene therapy, geneticist and journalist Lewis (Human Genetics) looks closely at the history of setbacks plaguing the treatment of rare genetic diseases as well as recent breakthroughs...Yet with each success, as Lewis recounts in this rigorous, energetic work, possibilities in treating HIV infection and dozens of other diseases might be around the next corner. Publisher's Weekly (starred review)

A fascinating account of groundbreaking science and the people who make it possible. Kirkus

Ricki Lewis gives us the inspiring story of gene therapy as told through Corey's eyes--literally. Her book delves into the challenges modern medicine faces--both in its bitter disappointments and great successes--but it goes much deeper than that. With empathy and grace, Lewis shows us the unimaginable strength of parents with sick children and the untiring devotion of the physicians who work to find the forever fix' to save them. But best of all Lewis gives us a story of profound hope. Molly Caldwell Crosby, author of The American Plague: The Untold Story of Yellow Fever, the Epidemic that Shaped Our History and Asleep: The Forgotten Epidemic that Remains One of Medicine's Greatest Mysteries

The Forever Fix is a wonderful story told by one of our most gifted science and medical writers. In the tradition of Siddhartha Mukherjee's The Emperor of All Maladies, Ricki Lewis explains complex biological processes in extremely understandable ways, ultimately providing crucial insights into the modeling of disease and illustrating how gene therapy can treat and even potentially cure the most challenging of our health conditions. Dennis A. Steindler, Ph.D., former Executive Director of the McKnight Brain Institute, University of Florida

Ricki Lewis has written a remarkable book that vividly captures the breathtaking highs and devastating lows of gene therapy over the past decade while giving ample voice to all sides -- the brave patient volunteers, their parents and physicians. The Forever Fix is required reading as we dare to dream of curing a host of genetic diseases. Kevin Davies, Founding editor of Nature Genetics; author of The $1,000 Genome and Cracking the Genome

In 'The Forever Fix,' Ms. Lewis chronicles gene therapy's climb toward the Peak of Inflated Expectations over the course of the 1990s. A geneticist and the author of a widely used textbook, she demonstrates a mastery of the history. The Wall Street Journal

An engaging and accessible look at gene therapy. Times Union

Medical writer Ricki Lewis interweaves science, the history of medical trial and error, and human stories from the death in 1999 of teenager Jesse Gelsinger, from a reaction to gene therapy intended to combat his liver disease, to radical successes in some children with adenosine deaminase deficiency. Nature

Lewis adeptly traverses the highs and lows of gene therapy and explores its past, present, and future through the tales of those who've tested its validity. The Scientist

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The Forever Fix: Gene Therapy and the Boy Who Saved It ...

Scientists have promised that gene therapy will be the next big leap for medicine. It's a term that's tossed about regularly, but what is it exactly? Trace shows us how scientists can change your very genetic code.

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How does gene therapy work?http://ghr.nlm.nih.gov/handbook/thera..."Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein."

Gene therapy trial 'cures children'http://www.bbc.co.uk/news/health-2326..."A disease which robs children of the ability to walk and talk has been cured by pioneering gene therapy to correct errors in their DNA, say doctors."

Gene therapy cures diabetic dogshttp://www.newscientist.com/article/d..."Five diabetic beagles no longer needed insulin injections after being given two extra genes, with two of them still alive more than four years later."

Gene Therapy for Cancer: Questions and Answershttp://www.cancer.gov/cancertopics/fa..."Gene therapy is an experimental treatment that involves introducing genetic material into a person's cells to fight or prevent disease."

How does gene therapy work?http://www.scientificamerican.com/art..."Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that's what viruses evolved to do with their own genetic material."

Gene therapy cures leukaemia in eight dayshttp://www.newscientist.com/article/m...eight-days.htmlWITHIN just eight days of starting a novel gene therapy, David Aponte's "incurable" leukaemia had vanished. For four other patients, the same happened within eight weeks, although one later died from a blood clot unrelated to the treatment, and another after relapsing.

Cell Therapy Shows Promise for Acute Type of Leukemiahttp://www.nytimes.com/2013/03/21/hea..."A treatment that genetically alters a patient's own immune cells to fight cancer has, for the first time, produced remissions in adults with an acute leukemia that is usually lethal, researchers are reporting."

Watch More:Tricking the Immune Systemhttp://www.youtube.com/watch?v=Kr_HRl...Babies with 3 Parents?!http://www.youtube.com/watch?v=jQxsW_...Pick Your Poison: Cyanidehttp://www.youtube.com/watch?v=JDBrdE...____________________

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How Does Gene Therapy Work? - YouTube

A retrovirus is any virus belonging to the viral family Retroviridae. All The genetic material in retroviruses is in the form of RNA molecules, while the genetic material of their hosts is in the form of DNA. When a retrovirus infects a host cell, it will introduce its RNA together with some enzymes into the cell. This RNA molecule from the retrovirus must produce a DNA copy from its RNA molecule before it can be considered part of the genetic material of the host cell. Retrovirus genomes commonly contain these three open reading frames that encode for proteins that can be found in the mature virus. Group-specific antigen (gag) codes for core and structural proteins of the virus, polymerase (pol) codes for reverse transcriptase, protease and integrase, and envelope (env) codes for the retroviral coat proteins (see figure 1). Figure 1. Genome organisation of retroviruses.

The process of producing a DNA copy from an RNA molecule is termed reverse transcription. It is carried out by one of the enzymes carried in the virus, called reverse transcriptase. After this DNA copy is produced and is free in the nucleus of the host cell, it must be incorporated into the genome of the host cell. That is, it must be inserted into the large DNA molecules in the cell (the chromosomes). This process is done by another enzyme carried in the virus called integrase (see figure 2).

Now that the genetic material of the virus is incorporated and has become part of the genetic material of the host cell, we can say that the host cell is now modified to contain a new gene. If this host cell divides later, its descendants will all contain the new genes. Sometimes the genes of the retrovirus do not express their information immediately.

Retroviral vectors are created by removal op the retroviral gag, pol, and env genes. These are replaced by the therapeutic gene. In order to produce vector particles a packaging cell is essential. Packaging cell lines provide all the viral proteins required for capsid production and the virion maturation of the vector. These packaging cell lines have been made so that they contain the gag, pol and env genes. Early packaging cell lines contained replication competent retroviral genomes and a single recombination event between this genome and the retroviral DNA vector could result in the production of a wild type virus. Following insertion of the desired gene into in the retroviral DNA vector, and maintainance of the proper packaging cell line, it is now a simple matter to prepare retroviral vectors (see figure 3).

One of the problems of gene therapy using retroviruses is that the integrase enzyme can insert the genetic material of the virus in any arbitrary position in the genome of the host. If genetic material happens to be inserted in the middle of one of the original genes of the host cell, this gene will be disrupted (insertional mutagenesis). If the gene happens to be one regulating cell division, uncontrolled cell division (i.e., cancer) can occur. This problem has recently begun to be addressed by utilizing zinc finger nucleases or by including certain sequences such as the beta-globin locus control region to direct the site of integration to specific chromosomal sites.

Gene therapy trials to treat severe combined immunodeficiency (SCID) were halted or restricted in the USA when leukemia was reported in three of eleven patients treated in the French X-linked SCID (X-SCID) gene therapy trial. Ten X-SCID patients treated in England have not presented leukemia to date and have had similar success in immune reconstitution. Gene therapy trials to treat SCID due to deficiency of the Adenosine Deaminase (ADA) enzyme continue with relative success in the USA, Italy and Japan.

As a reaction to the adverse events in the French X-SCID gene therapy trial, the Recombinant DNA Advisory Committee (RAC) sent a letter to Principal Investigators Conveying RAC Recommendations in 2003. In addition, the RAC published conclusions and recommendations of the RAC Gene Transfer Safety Symposium in 2005. A joint working party of the Gene Therapy Advisory Committee and the Committee on Safety of Medicines (CSM) in the UK lead to the publication of an updated recommendations of the GTAC/CSM working party on retroviruses in 2005.

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Gene Therapy Retrovirus Vectors Explained

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

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cancer: Gene therapy

Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy is to replace a defective gene with its normal counterpart, using methods of recombinant DNA technology. Methods to insert genes into

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Some have argued that gene therapy is eugenic but that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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Gene therapy | medicine | Britannica.com

Gene therapy delivers DNAinto a patients cells to replace faulty or missing genes or adds new genes in an attempt to cure diseases or to make changes so the body is better able tofight off disease. The DNA for a gene or genes is carried into a patientscells by a delivery vehicle called a vector, typically a specially engineeredvirus. The vector then inserts the gene(s) into the cells' DNA.

Although gene therapy is relativelynew and often still considered experimental, it can provide a cure for life-threateningdiseases that dont respond well to other therapies (includingimmunodeficiencies, metabolic disorders, and relapsed cancers) and for acuteconditions that currently rely on complex and expensive life-long medicationand management (such as sickle cell disease and hemophilia).

CAR T-Cell Therapy for Relapsed Acute Lymphoblastic Leukemia (ALL)

Dana-Farber/Boston Childrens is a certified treatment center for providing the recently-FDA-approved CAR T-cell therapy called KYMRIAH for relapsed B-cell acute lymphoblastic leukemia (ALL). This promising new treatment entails genetic engineering of the patients own T-cells to increase targeting of a specific leukemia protein and then accelerate killing of the target. After modification, they are returned to the patient via IV where they can immediately begin destroying circulating cancer cells.

For more information about CAR T-cell therapy, contact our gene therapy program

Our Gene Therapy Clinical Trials

Learn more about our gene therapy clinical trials

Dana-Farber/BostonChildrens has one the most extensive and long-running pediatric gene therapyprograms in the world. Since 2010, wehave treated 36 patients from 11 countries through eight gene therapy clinicaltrials.

Why choose Dana-Farber/BostonChildrens:

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