Adeno-associated virus as a delivery vector for gene therapy of human diseases

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods. Seven rAAV-based gene therapy products have received regulatory approval, but there continue to be concerns about safely using high-dose viral therapies in humans, including immune responses and adverse effects such as genotoxicity, hepatotoxicity,…

• AA
  American Australian Association
• NI
  National Institutes of Health

  Awards: R01HL097088, R01AI121135, R01NS076991, P01HL131471-05, UG3HL147367, R01HL152723, UH3HL147367-04, UG3HL147367-01, R01HL152723-02, R01NS076991-01, P01HL131471, U19AI149646-01
• DO
  Division of Graduate Education