Analysis of Adeno-Associated Virus (AAV) Capsid Proteins

What is Gene Therapy?

Gene therapy is an expanding area of medicine whereby specific medical conditions are treated at the DNA level through direct manipulation of the faulty gene. This has proven a viable approach through the use of viral vectors, serving as DNA delivery systems, that themselves have no negative pathogenicity to humans, whilst still being infectious and able to deliver the appropriate DNA. Conditions for which these gene therapy products are currently approved include spinal muscular atrophy and inherited retinal dystrophy with many, many more products under development.

What do the Gene Therapy regulations say about structural analysis?

What is the structure of the Adeno Associated Virus capsid?

What techniques are used to characterize AAV capsid proteins?