A Closer Look into Adeno-Associated Virus (AAV) Technology

One of the most exciting advances in modern medicine is the discovery of how the adeno-associated virus (AAV) can be used as an effective delivery system for therapeutic genetic material into living tissue. Having become the dominant form of gene therapy for genetic diseases, AAV is driving today’s therapeutic discoveries and is the most commonly used delivery system in FDA approved gene therapies.

new gene The replacement gene is transferred to the target cell via the AAV vector. viral vector vector bindsto cell vector packagedinto capsid vesicle breaksdown releasingvector new gene injectedinto nucleus nucleus cytoplasm

Main Benefits of Using AAV Technology

AAV is a virus that is not known to result in human disease

AAV will not replicate in the body like normal viruses do

The quantity of AAV delivery can be precisely controlled

AAV Delivery is Used to Treat Many Diseases

With broad therapeutic implications for a vast array of diseases, many companies have adopted AAV as the gene delivery method of choice for the treatments of diseases spanning from Leber Congenital Amaurosis, to Hemophilia B, and Spinal Muscular Atrophy.