In this webinar, Dr. Gao presents a comprehensive overview of the key principles, history, current challenges, and future directions of human gene therapy, with a focus on AAV gene therapy for rare diseases. The session also highlights AAV capsid engineering to modulate target tissue tropism and biodistribution, with examples of using AAV for gene replacement, gene addition, gene silencing, and in vivo gene editing in preclinical studies.

Additionally, Dr. Gao reviews strategies to overcome immunological barriers to enhance the efficacy of AAV-mediated gene therapy.