AAV (Adeno-Associated Virus)

Adeno-associated virus (AAV) is a small virus that is commonly used in gene therapy. Unlike many viruses, AAV does not cause diseases in humans. Scientists harness AAV to deliver therapeutic genes into specific cells to treat genetic disorders. By packaging the desired gene within the virus, it can safely enter cells and potentially correct genetic defects. AAV is favored for its ability to target various tissues and its safety profile, making it a promising tool for developing treatments for conditions like cystic fibrosis, hemophilia, and some forms of blindness.