AAV-adenine base editor targeting APOC3

AAV-adenine base editor targeting APOC3 (specifically identified as aioAAV-ABE-hAPOC3 in research) is an experimental gene therapy designed for the treatment of hypertriglyceridemia. Developed by researchers at the University of Michigan and Indiana University, this system employs an all-in-one adeno-associated virus (AAV) vector to deliver a compact adenine base editor (ABE). Unlike traditional CRISPR methods that create double-strand breaks, this base editing approach introduces precise A-to-G transitions within the human APOC3 gene to achieve permanent, liver-specific knockout. By reducing the levels of Apolipoprotein C3, a key regulator of triglyceride-rich lipoprotein metabolism, the therapy aims to durably lower plasma triglycerides and mitigate the risk of associated conditions such as acute pancreatitis and atherosclerotic cardiovascular disease.

Other names

aioABE-hAPOC3aioABE-hAPOC-3aioABE-hAPOC 3all-in-one AAV-ABE-hAPOC3