World’s First Gene Therapy Developed for Type 1 Diabetes

The Australian government has funded the Garvan Institute of Medical Research, an Australian biomedical research organization, to conduct the world’s first clinical trial of gene therapy for type 1 diabetes.

The treatment was developed thanks to a decade of $600 million ($385 million) from the government. Research Promotion Programintended to address chronic and complex diseases.

This is the first time genetically engineered pancreatic cells have been transplanted into humans to treat type 1 diabetes.

Gene therapy is an experimental technique that uses genes to treat or prevent disease instead of using drugs or surgery. It changes a person’s genetic makeup by delivering genetically engineered DNA molecules into the patient’s body.

Professor Shane Gray, principal investigator at the Garvan Institute, said clinical trials are the beginning of a long and exciting journey.

“We’ve been working on this research for 20 years now, and it’s amazing to see it reach the point where it’s translated into people,” Gray said in a release on October 31.

To manipulate cells, the researchers identified a key protein involved in inflammation and autoimmune disease known as A20 that can be used to regulate the immune system.

They discovered that the protein could be used to genetically engineer pancreatic islet cells to produce insulin.

“A20 is like a thermostat for your immune system. You can boil it down or raise it to make it more aggressive,” Gray said.

“For type 1 diabetes, it can be used as a handbrake for the immune system to stop damage to pancreatic islet cells.”

Gray and his team found that genetically engineered cells in a mouse model achieved 80-100% viability of pancreatic islet grafts without the need for heavy immunosuppressive drugs to keep the immune system from attacking the body. has been shown to have

“Genetically engineered cells seem to re-educate the immune system to accept the transplant as self. rice field.

New technology with unknown risks

Gene therapy has accelerated since the outbreak of COVID-19, with recent studies suggesting that gene therapy could be used to treat head and neck cancer, sickle cell anemia, rare neurological disorders, and congenital blindness as future treatments. are being considered for treatment as

However, despite the potential benefits of treatment, there are significant risk factors closely associated with gene therapy.

Osteopathic physician Joseph Mercola says that while gene therapy can reduce the symptoms of disease, it is an experimental technique that alters an individual’s genetic coding, turning a person into a “viral protein factory with no off-switch.”

According to, “Gene therapy is a promising treatment option for many diseases. [but] This technique is still dangerous and is still being researched to make sure it is safe and effective. Gene therapy is currently being tested only for diseases for which there are no other cures. ”

Jesse Chan


Jessie Zhang is a Sydney-based reporter covering Australian news with a focus on health and the environment. Please contact her at [email protected]