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Gene study shows possibility of patient-specific diabetes drugs
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Researchers from Weill Cornell Medicine have developed a method that uses human embryonic stem cells to show individual genetic mutations, which could be used to develop patient-specific drugs to treat diabetes, according to a press release from the institution.
Genomic studies have found that genetic mutations may contribute to diabetes.
Shuibing Chen, MD, assistant professor of chemical biology in surgery at Weill Cornell Medicine, and colleagues, studied three mutated genes — CDKAL1, KCNQ1 and KCNJ11I — by using stem cells that were directed to function as beta cells. The researchers found that mutations in these genes diminished the function of beta cells by decreasing insulin release and interfering with glucose regulation. Mutations in CDKAL1 also resulted in beta cells being highly sensitive to high blood glucose and high fat levels.
Researchers then screened 2,000 drugs and found one that corrects the CDKAL1; it is currently being studied in a phase 2 trial. The researcher hope that their modeling can lead scientists to develop therapies that target these genetic mutations.