AAV
AAV stands for adeno-associated virus. In genetic medicine, scientists often use engineered AAVs as vectors, or delivery vehicles, to carry genetic instructions into cells. The virus is modified so it is not used in the same way as a natural infection; its job is to help deliver a therapeutic payload. AAVs are important in rare disease because they can reach certain tissues, but they also have limits related to dose, immune response, manufacturing, repeat dosing, and how much genetic material they can carry.
Why it matters
Families often hear AAV discussed in gene therapy trials. Understanding it helps clarify why delivery, immune response, tissue targeting, and gene size can make or break a therapy program.
Related content
What Is Gene Therapy?
Gene therapy is a treatment approach that tries to address disease at the level of the gene, often by adding, replacing, silencing, or editing genetic instructions.
Dr. Guoping Feng (MIT) on SHANK3 Gene Therapy, Brain Disorders, and What’s Coming Next
Dr. Guoping Feng
Welcome — I’m Ron Kleiman, and this is GENEration Hope. In this episode, I’m joined by Dr. Guoping Feng, Professor of Neuroscience at MIT, affiliated with the McGovern Institute, the Yang Tan Collective, and the Broad Institute. 
What AI Drug Discovery Could Mean for Rare Disease Families
AI drug discovery is entering a new phase: not just better software, but a new industrial stack linking frontier models, pharma data, robotics, and real experiments.
Why it matters: Rare disease research often starts with small datasets, limited funding, urgent timelines, and difficult trial design. AI-linked discovery systems could help researchers generate stronger candidates and better experiments faster, but families should watch for clinical validation, access, manufacturing, and clear evidence rather than hype.
Source: GENEration Hope editorial analysis