Gene Therapy for Type 1 Diabetes: Preclinical Promise
Despite eclectic ways of delivering insulin to control blood glucose level in people with type 1 diabetes (T1D), no approach precisely replicates what happens in the body. Gene therapy may hold the answer.
T1D is usually autoimmune, with inherited risk factors such as certain HLA haplotypes contributing to, but not directly causing, the condition. A clever use of gene therapy is to commandeer liver cells to step in for the pancreatic beta cells that autoimmunity destroys.
MILESTONES IN HISTORY
One of the most classic stories of modern medicine concerns T1D: discovery of insulin at the University of Toronto in the early 1920s. Young surgeon Frederick Banting and medical student Charles Best were grudgingly given lab space and ten beagles to conduct their famous experiments that identified “isletin”.
In the summer of 1921, the hormone kept alive Marjorie, a dog whose pancreas had been removed, and in January 1922, it saved the first patient, 14-year-old Leonard Thompson.
Human insulin was the first drug produced using recombinant DNA technology. From shocking new biotechnology circa 1982, human insulin is today an item easily picked up at a drug store. But effectively using human insulin requires frequent monitoring and timing of injections, and even the most careful schedule sometimes can’t prevent long-term effects of uncontrolled blood glucose levels.
The prototype insulin pump dates to 1963; a wearable form came a decade later. Many people use pumps today to provide insulin in a manner more like a pancreas.
Thousands of islet transplants — the cell clusters that i Continue reading