Anti-Sam68 gene therapy for the treatment of type 2 diabetes, hypercholesterolemia, atherosclerosis, and obesity.

  • Adeno-associated virus (AAV)-based gene therapy targeting hepatic Sam68
  • Single therapy to treat type 2 diabetes (T2D), hypercholesterolemia and associated complications
  • Targeting Sam68 also helps with weight loss and the appearance of a lean phenotype


  • Src-associated-in-mitosis-of-68kDa (Sam68) is a member of the signal-transducer-and activator-of-RNA (STAR) family of RNA-binding proteins and functions as an adaptor proteinduring RNA processing.
  • In murine models of diabetes, targeted suppression of Sam68 in mouse hepatocytes protects against diet-induced obesity, reduces hepatic gluconeogenesis, ameliorates hyperglycemia (in dietary-induced and in genetic diabetes);
  • Targeted suppression of Sam68 in mouse hepatocytes using AAV-based delivery of specific Sam68 shRNA (i) alters the expression of genes that control cholesterol uptake and biosynthesis such as SREBP-2 and PCSK9, (ii) reduces serum levels of total cholesterol and triglycerides, and (iii) limits the progression of atherosclerosis.
  • Deletion of Sam68 promotes weight loss and a lean phenotype in mice via increasing thermogenesis, energy expenditure and adipose tissue browning.

Contact Information

Name: Karen F. Bernard


Phone: (205) 934-8826