The Roadmap to a cure

AAV acts like a microscopic delivery capsule, bringing a healthy copy of a gene directly into the cells that need it most.

AAV-Based Gene Augmentation Therapy is a form of gene therapy that uses Adeno-Associated Virus (AAV) - a small, non-pathogenic virus that has been modified to safely carry therapeutic genes as a delivery vehicle to deliver a healthy copy of a gene to replace or supplement a faulty/missing gene. The therapy is typically administered through injection (intravenous, intramuscular, or directly into affected tissue) where the AAV vectors travel to target cells. Once inside cells, the therapeutic genes begin producing the missing or defective protein. This approach essentially gives cells the "instructions" they need to make proteins they can't produce due to genetic mutations.