New progress in temporarily reversing diabetes gene therapy
January 17, 2018 Source: WuXi PharmaTech
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];On December 19, 2017, the US FDA approved Spark Therapeutics gene therapy to treat rare hereditary blindness. This initiative has spurred efforts to find new ways to repair defective genes or cells in a variety of diseases, including the treatment of diabetes. Scientists at the University of Pittsburgh School of Medicine recently announced that they have made significant progress in using gene therapy to reverse diabetes.
Type 1 diabetes occurs when the immune system erroneously destroys insulin-producing beta cells in the pancreas. In a mouse model of type 1 diabetes, the researchers demonstrated a gene therapy that converts alpha cells in the pancreas into fully functional beta cells. The technology was published in the journal Cell Stem Cell.
The team used adeno-associated virus (AAV) vector technology to secrete two proteins, Pdx1 and MafA, into the pancreas—a technique similar to the core technology of Spark Therapeutics' gene therapy. These two proteins can reprogram alpha cells into cells that produce insulin. The mice that used this technique maintained normal blood glucose levels for about four months.
One potential advantage of converting alpha cells to beta cells is that the alpha cells themselves are sufficiently visible that the immune system is unlikely to misidentify them as beta cells and attack them. The researchers compared gene expression patterns between normal beta cells and alpha cells transformed into insulin producers, confirming that these cells have completed "almost complete cell reprogramming."
Many new diabetes therapies are working to induce the immune system to work properly. For example, scientists at the Cardiff University School of Medicine in the UK are investigating a technique that uses proinsulin peptides to prevent destructive immune cells from attacking beta cells. Researchers at the Massachusetts General Hospital are testing tuberculosis vaccine BCG (BCG), which they found to restore regulatory T cells (Tregs), an immune cell that prevents beta cells from being destroyed. In November last year, a research team at Boston Children's Hospital published their findings, demonstrating that the enhanced immune checkpoint protein PD-L1 prevents insulin-secreting cells from being destroyed.
â–² Dr. George Gittes, lead author of the study (Source: University of Pittsburgh official website)
The study's lead author, Dr. George Gittes, said in a statement: "This study is the first to describe a single clinically convertible intervention in autoimmune diabetes that allows blood sugar to reach normal levels." In addition, this method does not inhibit the immune system, and suppressing the immune system is the main risk of autoimmune disease therapy.
Dr. Gittes and his colleagues point out that they still need to do more research to determine whether the efficacy of this therapy can be successfully transferred from mice to humans. Currently, they are testing this therapy in primates. And plans to seek approval from the US FDA to conduct clinical trials in patients with type 1 or type 2 diabetes.
Reference materials:
[1] Gene therapy temporary reverses diabetes in mice
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