The potential for treating type 1 diabetes

Many people who have recently been diagnosed with type 1 diabetes (T1D) immediately wonder, “When will there be a cure?” »

With the potential for a cure hanging in front of people with T1D for what seems like an eternity, more and more researchers currently believe that gene therapy could finally – one day soon, even – be the so-called “cure.” which has been so elusive.

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

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

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

For T1D, gene therapy might look like reprogramming alternative cells, causing these reprogrammed cells to perform the functions of your original insulin production. beta cells would work otherwise. If you have diabetes, this includes insulin production.

But the reprogrammed cells would be different enough from beta cells that your own immune system wouldn’t recognize them as “new cells” and attack them, which happens when T1D develops.

Although gene therapy is still in its infancy and is only available through clinical trials, the evidence to date is increasingly clear about the potential benefits of this treatment.

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

Alpha cells are the perfect cell type to transform into beta-like cells because not only are they also located in the pancreas, but they are abundant in your body and similar enough to beta cells for transformation to be possible. Beta cells produce insulin to lower your blood sugar level, while alpha cells produce glucagon, which raises your blood sugar level.

In the study, the mice’s blood sugar levels were normal for 4 months with gene therapy, all without immunosuppressive drugs, which inhibit or prevent the activity of your immune system. The newly created alpha cells, functioning like beta cells, were resistant to immune attacks from the body.

But the normal glucose levels seen in the mice were not permanent. This could potentially translate to several years of normal glucose levels in humans rather than a long standing cure.

In this Wisconsin study from 2013 (updated to 2017), researchers found that when a small DNA sequence was injected into the veins of diabetic rats, it created insulin-producing cells that normalized blood sugar levels by up to 6 weeks. All this 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.

Here’s how the study worked:

  • The injected DNA sequence detected an increase in glucose in the body.
  • Using a glucose-inducible response element, the injected DNA began to produce insulin, similar to how beta cells produce insulin in a functioning pancreas.

Researchers are now working to increase the time interval between therapeutic 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 convenient the therapy is for the people. Ultimately, the hope is that AAV vectors could eventually be delivered to the pancreas through a non-surgical endoscopic procedure, in which a doctor uses a medical device with a light attached to look inside your body.

These types of gene therapy would not be a one-size-fits-all cure. But it would be a great relief for people with diabetes to enjoy perhaps several years of diabetes-free glucose levels without taking insulin.

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

Does it count as a cure?

It all depends on who you ask, as the definition of “a cure” for T1D varies.

Some people believe that a cure is a one-off business. For them, a “cure” means you never have to think about taking insulin, checking your blood sugar or the ups and downs of diabetes ever again. It even means you never have to go back to the hospital for follow-up gene therapy treatment.

Other people believe that a gene-editing treatment once every few years may be a sufficient treatment plan to be considered a cure.

Many others believe that you have to repair the underlying autoimmune response to be truly “cured”, and some people don’t care one way or another, as long as their blood sugar is normal and the mental burden of diabetes is relieved.

A 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 body’s DNA, and if you have type 1 diabetes, the idea is to tackle the underlying cause of the autoimmune attack. that destroyed your beta cells and caused T1D.

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

The goal of gene editing is simply to remove the bad parts of our DNA in order to completely avoid conditions such as diabetes and stop the ongoing immune response (attack on beta cells) that people who already have diabetes suffer daily (unbeknownst to them). ).

Gene editing performed by CRISPR through its partnership with ViaCyte creates insulin-producing islet cells capable of escaping an autoimmune response. These technologies and research are constantly evolving and are very promising.

Additionally, a 2017 study shows that a J1Dvsure may one day be possible using gene editing technology.

Gene therapy and gene editing hold great promise for people living with T1D who hope for an eventual future without the need for insulin or immunosuppressive therapy.

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

While gene therapy and gene editing therapy are still in their infancy (and many have been delayed by coronavirus disease 19 [COVID-19] pandemic), there is great hope for a cure of T1D in the near future.

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