AAV-Hnf1α

AAV-Hnf1α is an investigational pancreas-directed gene therapy designed for the treatment of maturity-onset diabetes of the young type 3 (MODY3). Developed by researchers at the Universitat Autònoma de Barcelona (UAB) in collaboration with CIBERDEM and the Universitat de Vic, the therapy utilizes adeno-associated viral (AAV) vectors to deliver the hepatocyte nuclear factor 1A (Hnf1α) gene under the control of a β-cell-specific promoter. Administered via intra-pancreatic duct delivery, AAV-Hnf1α aims to restore Hnf1α expression specifically in pancreatic β-cells, thereby upregulating key target genes such as GLUT2 and L-PK. In preclinical models of MODY3, the therapy has been shown to counteract hyperglycemia, improve glucose tolerance, and enhance insulin secretion, potentially offering a disease-modifying treatment that addresses the root genetic cause of the disease.

Other names

AAV-Hnf1aAAV-Hnf-1aAAV-Hnf 1a