AAV9-APC minigene

AAV9-APC minigene is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed to restore the function of the adenomatous polyposis coli (APC) tumor suppressor. Mutations in the APC gene, which lead to truncated proteins and dysregulated β-catenin degradation, are primary drivers of colorectal neoplasia and familial adenomatous polyposis (FAP). Because the full-length APC gene is too large for standard AAV packaging, this therapy utilizes a 'minigene' construct containing essential β-catenin and Axin (SAMP) binding sites to scaffold the β-catenin destruction complex. Developed by researchers at the Medical University of South Carolina, the therapy aims to reduce Wnt signaling, promote β-catenin ubiquitination, and inhibit polyp formation and growth in the colon. Preclinical data has demonstrated that systemic administration of the AAV9-APC minigene can successfully transduce colonic epithelium and significantly reduce polyp size and number in mouse models.

Other names

APC minigeneAAV9-APCAAV-9-APCAAV 9-APC