Category Archives: Gene therapy

gene editing, the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. Gene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA. Key among gene-editing technologies is a molecular tool known as CRISPR-Cas9, a powerful technology discovered in 2012 by American scientist Jennifer Doudna, French scientist Emmanuelle Charpentier, and colleagues and refined by American scientist Feng Zhang and colleagues. CRISPR-Cas9 functioned with precision, allowing researchers to remove and insert DNA in the desired locations.

The significant leap in gene-editing tools brought new urgency to long-standing discussions about the ethical and social implications surrounding the genetic engineering of humans. Many questions, such as whether genetic engineering should be used to treat human disease or to alter traits such as beauty or intelligence, had been asked in one form or another for decades. With the introduction of facile and efficient gene-editing technologies, particularly CRISPR-Cas9, however, those questions were no longer theoretical, and the answers to them stood to have very real impacts on medicine and society.

The idea of using gene editing to treat disease or alter traits dates to at least the 1950s and the discovery of the double-helix structure of DNA. In the mid-20th-century era of genetic discovery, researchers realized that the sequence of bases in DNA is passed (mostly) faithfully from parent to offspring and that small changes in the sequence can mean the difference between health and disease. Recognition of the latter led to the inescapable conjecture that with the identification of molecular mistakes that cause genetic diseases would come the means to fix those mistakes and thereby enable the prevention or reversal of disease. That notion was the fundamental idea behind gene therapy and from the 1980s was seen as a holy grail in molecular genetics.

The development of gene-editing technology for gene therapy, however, proved difficult. Much early progress focused not on correcting genetic mistakes in the DNA but rather on attempting to minimize their consequence by providing a functional copy of the mutated gene, either inserted into the genome or maintained as an extrachromosomal unit (outside the genome). While that approach was effective for some conditions, it was complicated and limited in scope.

In order to truly correct genetic mistakes, researchers needed to be able to create a double-stranded break in DNA at precisely the desired location in the more than three billion base pairs that constitute the human genome. Once created, the double-stranded break could be efficiently repaired by the cell using a template that directed replacement of the bad sequence with the good sequence. However, making the initial break at precisely the desired locationand nowhere elsewithin the genome was not easy.

Before the advent of CRISPR-Cas9, two approaches were used to make site-specific double-stranded breaks in DNA: one based on zinc finger nucleases (ZFNs) and the other based on transcription activator-like effector nucleases (TALENs). ZFNs are fusion proteins composed of DNA-binding domains that recognize and bind to specific three- to four-base-pair-long sequences. Conferring specificity to a nine-base-pair target sequence, for example, would require three ZFN domains fused in tandem. The desired arrangement of DNA-binding domains is also fused to a sequence that encodes one subunit of the bacterial nuclease Fok1. Facilitating a double-stranded cut at a specific site requires the engineering of two ZFN fusion proteinsone to bind on each side of the target site, on opposite DNA strands. When both ZFNs are bound, the Fok1 subunits, being in proximity, bind to each other to form an active dimer that cuts the target DNA on both strands.

TALEN fusion proteins are designed to bind to specific DNA sequences that flank a target site. But instead of using zinc finger domains, TALENs utilize DNA-binding domains derived from proteins from a group of plant pathogens. For technical reasons TALENs are easier to engineer than ZFNs, especially for longer recognition sites. Similar to ZFNs, TALENs encode a Fok1 domain fused to the engineered DNA-binding region, so, once the target site is bound on both sides, the dimerized Fok1 nuclease can introduce a double-stranded break at the desired DNA location.

Unlike ZFNs and TALENs, CRISPR-Cas9 uses RNA-DNA binding, rather than protein-DNA binding, to guide nuclease activity, which simplifies the design and enables application to a broad range of target sequences. CRISPR-Cas9 was derived from the adaptive immune systems of bacteria. The acronym CRISPR refers to clustered regularly interspaced short palindromic repeats, which are found in most bacterial genomes. Between the short palindromic repeats are stretches of sequence clearly derived from the genomes of bacterial pathogens. Older spacers are found at the distal end of the cluster, and newer spacers, representing more recently encountered pathogens, are found near the proximal end of the cluster.

Transcription of the CRISPR region results in the production of small guide RNAs that include hairpin formations from the palindromic repeats linked to sequences derived from the spacers, allowing each to attach to its corresponding target. The RNA-DNA heteroduplex formed then binds to a nuclease called Cas9 and directs it to catalyze the cleavage of double-stranded DNA at a position near the junction of the target-specific sequence and the palindromic repeat in the guide RNA. Because RNA-DNA heteroduplexes are stable and because designing an RNA sequence that binds specifically to a unique target DNA sequence requires only knowledge of the Watson-Crick base-pairing rules (adenine binds to thymine [or uracil in RNA], and cytosine binds to guanine), the CRISPR-Cas9 system was preferable to the fusion protein designs required for using ZFNs or TALENs.

A further technical advance came in 2015, when Zhang and colleagues reported the application of Cpf-1, rather than Cas9, as the nuclease paired with CRISPR to achieve gene editing. Cpf-1 is a microbial nuclease that offers potential advantages over Cas9, including requiring only one CRISPR guide RNA for specificity and making staggered (rather than blunt) double-stranded DNA cuts. The altered nuclease properties gave potentially greater control over the insertion of replacement DNA sequences than was possible with Cas9, at least in some circumstances. Researchers suspect that bacteria house other genome-editing proteins as well, the evolutionary diversity of which could prove valuable in further refining the precision and versatility of gene-editing technologies.

See more here:
Gene editing | Definition, History, & CRISPR-Cas9 | Britannica

As early as the 1960s, scientists speculated that DNA sequences could be introduced into patients cells to cure genetic disorders. In the early 1980s, David Williams, MD, and David Nathan, MD, at Boston Childrens Hospital published the first paper showing one could use a virus to insert genes into blood-forming stem cells. In 2003, the Human Genome Project wrapped up, giving us a complete blueprint of our DNA. In the past decade, gene therapy has become a reality for multiple diseases, especially those caused by mutations in a single gene.

Gene therapy falls into two main categories. Ex vivo gene therapy removes cells from the patient, introduces new genetic material, packaged in a delivery vehicle called a vector, then returns the cells to the patient. Boston Childrens is using this method for such disorders as sickle cell disease, adrenoleukodystrophy, chronic granulomatous disease and others. In vivo gene therapy involves direct IV infusion of the vector into the bloodstream or injection into a target organ like the eye. Boston Childrens uses in vivo gene therapy for several disorders, including hemophilia and ornithine transcarbamylase deficiency.

After a rocky start, gene therapy is on fire, drawing keen interest from the biopharmaceutical industry. And its still evolving and improving.

In 1990, 4-year-old Ashanthi de Silva became the first gene therapy success story. She was born with a severe combined immunodeficiency (SCID) due to lack of the enzyme adenosine deaminase (ADA). Without ADA, her T cells died off, leaving her unable to fight infections. Injections of a synthetic ADA enzyme helped, but only temporarily.

Doctors decided to deliver a healthy ADA gene into her blood cells, using a disabled virus that cannot spread in the body. Their success spurred more trials in the 1990s for the same form of SCID. Now in her 30s, de Silva is active in the rare disease community.

European researchers in the 1990s focused on SCID-X1, another form of SCID linked to the X chromosome. They reported the first cures in 2000, but within several years, five of the 20 treated children developed cancer.The viral vector that delivered the gene to their T cells had also activated an oncogene, triggering leukemia.

The U.S. saw another early setback: the 1999 death of 18-year-old Jesse Gelsinger, after receiving gene therapy for a rare metabolic disorder. In his case, the viral vector caused a fatal immune response.

Gene therapy came to a halt.

In the early 2010s, gene therapy experienced a renaissance. Scientists developed better viral vectors to deliver genetic therapies. They added regulatory elements called promoters and enhancers to direct the genes activity. These elements specified where and when the gene should turn on, and at what level. Investigators at Boston Childrens, in a global collaborative effort, led work that addressed the problem of leukemia, allowing gene therapy to resume for SCID-X1.

A REBIRTH IN BOSTON: GENE THERAPY TURNS 10

Born in 2010 with X-linkedsevere combined immunodeficiency(SCID-X1), Agustn spent the first few months of his life in isolation. He became the first patient to receive gene therapy at Boston Childrens and today is an active fifth-grade soccer and tennis player.

The new, modified vectors can more precisely target expression of genes in specific cell types, dont go astray in the body, and dont trigger the immune system. Some deliver genes meant to work for a short while and then inactivate themselves. Others carry genes that remain active long-term and pass to daughter cells as the cells divide. Popular viruses for gene therapy include adenoviruses, adeno-associated virus, and lentiviruses.

An example of an improved vector is the lentivirus vector used for sickle cell gene therapy at Boston Childrens. The vector silences a gene calledBCL11A, leading to production of fetal hemoglobin that is not affected by the sickle cell mutation. It was precision engineered to silence the gene only in precursors of red blood cells, a tweak that enabled the treated blood stem cells to live long-term in patients bone marrow. Williams led the vectors development, based on seminal research by Vijay Sankaran, MD, PhD, and Stuart Orkin, MD, in the Hematology/Oncology Program at Boston Childrens.

Traditional gene therapyuses viruses to carry healthy genes into cells, compensating for a faulty or missing gene. But the past decade has seen an explosion of other methods for delivering or fixing genes.

Gene editing uses various molecular tools that precisely target problematic genes and create a cut or break in their DNA. It can knock out a faulty gene, insert a new DNA sequence, or both in a cut and paste operation. The best-known gene editing systems are CRISPR/Cas 9, zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs). The next generation of gene therapy for sickle cell disease is utilizing CRISPR to edit the BCL11A gene, based on work by Dan Bauer, MD, PhD, at Boston Childrens, then in Orkins laboratory.

Base editingis even more fine-tuned. It leverages the targeting ability of CRISPR, but relies on enzymes to chemically change one letter of a genes code at a time changing, say, C to T or A to G. These small changes can correct a spelling error mutation, silence a disease-causing gene, or help activate a specific gene. Unlike gene editing, base editing hasnt yet been tested in clinical trials, but it offers the promise of more precision, efficiency, and safety.Boston Childrens has several base editing projects on deck.

Other new approaches blur the line between gene therapy and drug treatment. For example, antisense oligonucleotides (ASOs) are drugs made up of short, synthetic pieces of DNA or RNA that target the messenger RNA made by the faulty gene. They prevent the gene from being translated into a bad protein or, in some cases, trick the cells machinery into making a good protein. Researchers can even customize ASOs to single patients. Tim Yu, MD, PhD, in the Division of Genetics and Genomics at Boston Childrens, has developed this approach to treat several very rare genetic conditions.

Another approach, RNA interference, uses small RNAs to silence a targeted gene by neutralizing the genes mRNA. (The lentivirus described above uses RNA interference to silence the BCL11A gene.)

Even the messenger RNAs used for some COVID-19 vaccines represent a form of gene therapy. The mRNAs introduce genetic code that cells then use to make the coronavirus spike protein, encouraging people to develop antibodies to the virus.

Today, ClinicalTrials.gov lists nearly 400 active gene therapy studies all over the world, and more than a dozen gene therapy drugs are on the market. At Boston Childrens, the Gene Therapy Program has more than 20 human trials completed or underway, with more in the pipeline. While gene therapies are currently expensive, its expected that prices will come down over time. And as a one-time treatment, gene therapy promises to save money in the long run by preventing a lifetime of illness a true revolution in medicine.

Learn more about the Gene Therapy Program at Boston Childrens Hospital

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A short history of gene therapy - Boston Children's Answers

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. As the field strives to strike a delicate balance between safety and efficacy, in the context of increased regulatory scrutiny and safety challenges, attending the 4th Annual Gene Therapy Analytical Development as an analytical scientist has never been so important.

This years summit returns in-person to Boston to reunite 300+ analytical experts in innovative biotech, pharma and academia to continue to develop resilient, long-lasting and robust analytical tools to enhance the safety, quality and efficacy of gene therapy products.

Whether you are focusing on specific characterization methods, enhancing your genome sequencing, advancing your understanding of full and partial particles, or advancing your early-stage bioassays, with 4 tracks, 8 pre-conference workshops and a post-conference focus day, the 4th Gene Therapy Analytical Development Summit will encompass all aspects of analytical development, giving you the chance to address and overcome challenges.

If you work in quality control, quality assurance, or process development - weve listened and weve answered. This years agenda includes a novel track designed for quality control and process development groups working in gene therapy. Talks include enhancing the knowledge transfer between departments, bridging between analytical methods with regards to QC/PD, and enhancing in-process development support.

Whether you're working with AAV, non-viral vectors or lentiviral vectors, this is your opportunity to enhance your existing analytical methods and explore innovative new tools to support safe and effective gene therapy development.

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Gene Therapy Analytical Development Summit 2022 | Home

1. $3.5-Million Hemophilia Gene Therapy Is World's Most Expensive Drug  Scientific American
2. The Era of One-Shot, Multimillion-Dollar Genetic Cures Is Here  WIRED
3. The most expensive drug in the world: Hemgenix, a $3.5 million treatment for hemophilia B  EL PAS USA
4. 18-Year-Old Patient Says $3.5 Million Hemophilia Drug He Needs Seems a "Little Steep"  Futurism
5. View Full Coverage on Google News

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$3.5-Million Hemophilia Gene Therapy Is World's Most Expensive Drug - Scientific American

CAR T Global Consultant Inc. Announce their Collaboration with Titronbio - a company founded in Shanghai China by a renowned leader in the field of CAR T and Cell and Gene therapy  PR Newswire

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CAR T Global Consultant Inc. Announce their Collaboration with Titronbio - a company founded in Shanghai China by a renowned leader in the field of...

Cell and Gene Therapy Manufacturing Services Market Size In 2023 | Financial Performance, In-Depth Insight of Trends, Key Players (Thermo Fisher Scientific, Merck KGaA, Charles River Laboratories, Lonza), SWOT Analysis, Distributors/Traders List, Latest I  Digital Journal

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Cell and Gene Therapy Manufacturing Services Market Size In 2023 | Financial Performance, In-Depth Insight of Trends, Key Players (Thermo Fisher...

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The genes in your bodys cells play a key role in your health. Indeed, a defective gene or genes can make you sick.

Recognizing this, scientists have worked for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure, or prevent a disease or medical condition.

This research is paying off, as advancements in science and technology today are changing the way we define disease, develop drugs, and prescribe treatments.

The U.S. Food and Drug Administration has approved multiple gene therapy products for cancer and rare disease indications.

Genes and cells are intimately related. Within the cells of our bodies, there are thousands of genes that provide the information to produce specific proteins that help make up the cells. Cells are the basic building blocks of all living things; the human body is composed of trillions of them.

The genes provide the information that makes different cells do different things. Groups of many cells make up the tissues and organs of the body, including muscles, bones, and blood. The tissues and organs in turn support all our bodys functions.

Sometimes the whole or part of a gene is defective or missing from birth. This is typically referred to as a genetically inherited mutation.

In addition, healthy genes can change (mutate) over the course of our lives. These acquired mutations can be caused by environmental exposures. The good news is that most of these genetic changes (mutations) do not cause disease. But some inherited and acquired mutations can cause developmental disorders, neurological diseases, and cancer.

Depending on what is wrong, scientists can do one of several things in gene therapy:

To insert new genes directly into cells, scientists use a vehicle called a vector. Vectors are genetically engineered to deliver the necessary genes for treating the disease.

Vectors need to be able to efficiently deliver genetic material into cells, and there are different kinds of vectors. Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells. Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.

Gene therapy can be used to modify cells inside or outside the body.When a gene therapy is used to modify cells inside the body, a doctor will inject the vector carrying the gene directly into the patient.

When gene therapy is used to modify cells outside the body, doctors take blood, bone marrow, or another tissue, and separate out specific cell types in the lab. The vector containing the desired gene is introduced into these cells. The cells are later injected into the patient, where the new gene is used to produce the desired effect.

Before a gene therapy can be marketed for use in humans, the product must be tested in clinical studies for safety and effectiveness so FDA scientists can consider whether the risks of the therapy are acceptable considering the potential benefits.

The scientific field for gene therapy products is fast-paced and rapidly evolving ushering in a new approach to the treatment of vision loss, cancer, and other serious and rare diseases. As scientists continue to make great strides in this therapy, the FDA is committed to helping speed up development by interacting with those developing products and through prompt review of groundbreaking treatments that have the potential to save lives.

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How Gene Therapy Can Cure or Treat Diseases | FDA

1. FDA Approves First Gene Therapy to Treat Adults with Hemophilia B  FDA.gov
2. Gene therapy at $3.5m a dose approved for US adults with hemophilia B  The Guardian
3. FDA approves gene therapy for hemophilia  Axios
4. FDA Approves Hemgenix, First Gene Therapy to Treat Adults with Hemophilia B  Everyday Health
5. Costing $3.5M, first hemophilia B gene therapy wins FDA approval  FiercePharma
6. View Full Coverage on Google News

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FDA Approves First Gene Therapy to Treat Adults with Hemophilia B - FDA.gov

Vaccines that use mRNA technology are not gene therapy because they do not alter your genes, experts have told Reuters after contrary claims were posted online.

Thousands of social media users have shared such posts since the rollout of COVID-19 vaccines began (here) and have continued to do so through August.

Its not a vaccine. Its gene therapy! wrote one Facebook user on Aug. 9, noting that gene therapy manipulates genetic code (here and here).

Pfizer/BioNTech and Moderna have both developed shots that use a piece of genetic code from SARS-CoV-2, the coronavirus that causes COVID-19, to prompt an immune response in recipients (here). However, experts told Reuters that this is not the same as gene therapy.

As mRNA is genetic material, mRNA vaccines can be looked at as a genetic-based therapy, but they are classified as vaccines and are not designed to alter your genes, said Dr Adam Taylor, a virologist and research fellow at the Menzies Health Institute, Queensland, Griffith University.

Gene therapy, in the classical sense, involves making deliberate changes to a patients DNA in order to treat or cure them. mRNA vaccines will not enter a cells nucleus that houses your DNA genome. There is zero risk of these vaccines integrating into our own genome or altering our genetic makeup.

Taylor explained that mRNA enters cells shortly after vaccination and instructs them to create a SARS-CoV-2 spike protein, prompting the immune response.

He added that unlike gene therapy, mRNA vaccines are then rapidly degraded by the body.

In fact, because mRNA is degraded so quickly chemical modifications can be made to mRNA vaccines to make them a little more stable than regular mRNA.

Gene therapy, on the other hand, involves a process whereby an individuals genetic makeup is deliberately modified to cure or treat a specific genetic condition (here).

It can be done in several ways, such as replacing a disease-causing gene with a healthy alternative, disabling a disease-causing gene or introducing a new gene to help treat a disease, according to the U.S. Food and Drug Administration (FDA) (here).

If we suffer from an inherited blood disease then the defect in our genes can be corrected in blood cells and then we can be cured, said David Schaffer, professor of Chemical and Biomolecular Engineering and Director of the Berkeley Stem Cell Center at the University of California, Berkeley, in an email to Reuters.

In most cases, the DNA is therapeutic because it encodes a mRNA, which encodes a protein that has a beneficial effect on a patient. So, if someone has a disease where the gene encoding an important protein is mutated - such as hemophilia, cystic fibrosis, retinitis pigments - then it can be possible to deliver the DNA encoding the correct copy of that protein in order to treat the disease.

He added: Because DNA has the potential to persist in the cells of a patient for years, this raises the possibility of a single gene therapy treatment resulting in years of therapeutic benefit.

Moderna, which has developed one of the mRNA COVID-19 vaccines used across the world, explained in a fact sheet that mRNA and gene therapy take fundamentally different approaches.

Gene therapy and gene editing alter the original genetic information each cell carries, the company writes. The goal is to produce a permanent fix to the underlying genetic problem by changing the defective gene. Moderna is taking a different approach to address the underlying cause of MMA and other diseases. mRNA transfers the instructions stored in DNA to make the proteins required in every living cell. Our approach aims to help the body make its own missing or defective protein (www.modernatx.com/about-mrna).

Reuters has in the past debunked claims that COVID-19 vaccines can genetically modify humans here and here .

Missing context. Scientists told Reuters that while mRNA vaccines can be considered genetic-based therapy because they use genetic code from COVID-19, they are not technically gene therapy. This is because the mRNA does not change the bodys genetic makeup.

This article was produced by the Reuters Fact Check team. Read more about our work to fact-check social media postshere .

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Fact Check-mRNA vaccines are distinct from gene therapy ... - Reuters

Many people whove recently received a diagnosis of type 1 diabetes (T1D) immediately think, When will there be a cure?

While the potential for a cure has been dangling in front of people with T1D for what seems like forever, more researchers currently believe that gene therapy could finally one day soon, even be the so-called cure thats been so elusive.

This article will explain what gene therapy is, how its similar to gene editing, and how gene therapy could potentially be the cure for T1D, helping millions of people around the world.

Gene therapy is a medical field of study that focuses on the genetic modification of human cells to treat or sometimes even cure a particular disease. This happens by reconstructing or repairing defective or damaged genetic material in your body.

This advanced technology is only in the early research phases of clinical trials for treating diabetes in the United States. Yet, it has the potential to treat and cure a wide range of other conditions beyond just T1D, including AIDS, cancer, cystic fibrosis (a disorder that damages your lungs, digestive tract, and other organs), heart disease, and hemophilia (a disorder in which your blood has trouble clotting).

For T1D, gene therapy could look like the reprogramming of alternative cells, making those reprogrammed cells perform the functions your original insulin-producing beta cells would otherwise perform. If you have with diabetes, that includes producing insulin.

But the reprogrammed cells would be different enough from beta cells so that your own immune system wouldnt recognize them as new cells and attack them, which is what happens in the development of T1D.

While gene therapy is still in its infancy and available only in clinical trials, the evidence so far is becoming clearer about the potential benefits of this treatment.

In a 2018 study, researchers engineered alpha cells to function just like beta cells. They created an adeno-associated viral (AAV) vector to deliver two proteins, pancreatic and duodenal homeobox 1 and MAF basic leucine zipper transcription factor A, to a mouses pancreas. These two proteins help with beta cell proliferation, maturation, and function.

Alpha cells are the ideal type of cell to transform into beta-like cells because not only are they also located within the pancreas, but theyre abundant in your body and similar enough to beta cells that the transformation is possible. Beta cells produce insulin to lower your blood sugar levels while alpha cells produce glucagon, which increases your blood sugar levels.

In the study, mouse blood sugar levels were normal for 4 months with gene therapy, all without immunosuppressant drugs, which inhibit or prevent the activity of your immune system. The newly created alpha cells, performing just like beta cells, were resistant to the bodys immune attacks.

But the normal glucose levels observed in the mice werent permanent. This could potentially translate into several years of normal glucose levels in humans rather than a longtime cure.

In this Wisconsin study from 2013 (updated as of 2017), researchers found that when a small sequence of DNA was injected into the veins of rats with diabetes, it created insulin-producing cells that normalized blood glucose levels for up to 6 weeks. That was all from a single injection.

This is a landmark clinical trial, as it was the first research study to validate a DNA-based insulin gene therapy that could potentially one day treat T1D in humans.

This was how the study worked:

The researchers are now working on increasing the time interval between therapy DNA injections from 6 weeks to 6 months to provide more relief for people with T1D in the future.

While this is all very exciting, more research is needed to determine how practical the therapy is for people. Eventually, the hope is that the AAV vectors could eventually be delivered to the pancreas through a nonsurgical, endoscopic procedure, in which a doctor uses a medical device with a light attached to look inside your body.

These kinds of gene therapy wouldnt be a one-and-done cure. But it would provide a lot of relief to people with diabetes to perhaps enjoy several years of nondiabetes glucose numbers without taking insulin.

If subsequent trials in other nonhuman primates are successful, human trials may soon begin for the T1D treatment.

Does that count as a cure?

It all depends on who you ask because the definition of a cure for T1D varies.

Some people believe that a cure is a one-and-done endeavor. They see a cure as meaning youd never have to think about taking insulin, checking blood sugars, or the highs and lows of diabetes ever again. This even means you wouldnt have to ever go back to a hospital for a gene therapy follow-up treatment.

Other people think that a once-in-a-few-years treatment of gene editing may be enough of a therapy plan to count as a cure.

Many others believe that you need to fix the underlying autoimmune response to truly be cured, and some people dont really care one way or another, as long as their blood sugars are normal, and the mental tax of diabetes is relieved.

One potential one-and-done therapy could be gene editing, which is slightly different from gene therapy.

The idea behind gene editing is to reprogram your bodys DNA, and if you have type 1 diabetes, the idea is to get at the underlying cause of the autoimmune attack that destroyed your beta cells and caused T1D to begin with.

Two well-known companies, CRISPR Therapeutics and regenerative med-tech company ViaCyte, have been collaborating for a few years to use gene editing to create islet cells, encapsulate them, and then implant them into your body. These protected, transplanted islet cells would be safe from an immune system attack, which would otherwise be the typical response if you have T1D.

The focus of gene editing is to simply cut out the bad parts of our DNA in order to avoid conditions such as diabetes altogether and to stop the continuous immune response (beta cell attack) that people who already have diabetes experience daily (without their conscious awareness).

The gene editing done by CRISPR in their partnership with ViaCyte is creating insulin-producing islet cells that can evade an autoimmune response. These technology and research are ever evolving and hold a lot of promise.

Additionally, a 2017 study shows that a T1Dcure may one day be possible by using gene-editing technology.

Both gene therapy and gene editing hold a lot of promise for people living with T1D who are hoping for an eventual future without needing to take insulin or immunosuppressant therapy.

Gene therapy research continues, looking at how certain cells in the body could be reprogrammed to start making insulin and not experience an immune system response, such as those who develop T1D.

While gene therapy and gene-editing therapy are still in their early stages (and much has been held up by the coronavirus disease 19 [COVID-19] pandemic), theres a lot of hope for a T1D cure in our near future.

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Gene therapy: The Potential for Treating Type 1 Diabetes - Healthline