Novel gene therapy reverses type 1 diabetes in mice

Medical News on January 9th, US researchers said on the 4th that a new intra-pancreatic gene therapy has successfully restored blood glucose levels in type 1 diabetic mice to normal for a long period of time, and the therapy may have broad prospects.

Type 1 diabetes is an autoimmune disease that occurs in children and adolescents. The patient's own immune system mistakenly attacks and destroys insulin-producing beta cells. One treatment for this disease is to protect and restore functional beta cells, but beta cell replacement therapy is not destined to succeed because the new cells injected are also attacked by the immune system.

Researchers at the University of Pittsburgh, USA, use a novel gene therapy approach to deliver an adeno-associated viral vector carrying two recoded genes, Pdx1 and MafA, through the pancreatic duct to the pancreas of type 1 diabetic mice, transforming alpha cells into A functional beta-like cell that secretes insulin.

Published in a new issue of the US Cell-Stem Cell Journal, the neonatal beta-like cells produced in this therapy are slightly different from native beta cells and are relatively resistant to autoimmune attacks. After a single treatment, blood glucose in type 1 diabetic mice returned to normal levels and lasted for up to 4 months. After 4 months, the mice resumed hyperglycemia, indicating that this gene therapy is still unable to cure type 1 diabetes.

Xiao Xiangyu, one of the paper leaders and assistant professor at the University of Pittsburgh, told Xinhua News Agency: "This method is expected to be applied to clinical trials in patients with type 1 and type 2 diabetes for the foreseeable future, because the pancreatic duct pancreas used in this study Gene editing technology has a corresponding therapeutic approach for patients."

At present, the research team has carried out similar experiments in non-human primates and obtained better therapeutic effects. Xiao Xiangxi said that if the treatment effect is confirmed, the research team will seek approval from the US regulatory authorities to prepare clinical trials of this gene therapy in patients with type 1 and type 2 diabetes.