From Concept To Cure: Using AAV In Gene Therapy

In gene therapy, Adeno-Associated Virus (AAV) vectors are the preferred method for treating or preventing disease due to their low immunogenicity. However, these vectors can still trigger immune responses in patients. To manage this, clinicians screen for pre-existing antibodies against AAV serotypes or temporarily clear antibodies from the patient's blood.

Over fifty clinical trials are currently being conducted to develop treatments for rare genetic disorders. The article highlights how Bio-Rad's ChemiDoc MP Imaging System can be used in the quality control process of recombinant AAV (rAAV) assembly and production. This system can identify AAV capsid surface protein types, assembled rAAV VP ratio, and final gene products. Despite challenges, AAV-based therapeutics have shown potential in curing genetic diseases.