Opinion
Video
Overview of Gene Therapy Approaches and Impacts
Experts delve into the fundamentals of gene therapy, covering in vivo and ex vivo methods, and explore cellular function targets for effective treatment of a disease.
This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.
Ciafaloni offers a comprehensive overview of gene therapy, highlighting its 2 primary approaches: in vivo and ex vivo. She elaborates on the 4 basic gene therapy approaches, emphasizing the significance of targeting specific cells and tissues based on disease mechanisms. From replacing faulty genes to silencing or editing them, the techniques vary in their application depending on the cellular context and disease pathology. The dialogue underscores the broad spectrum of gene therapy’s potential and the importance of optimizing delivery mechanisms for its continued innovation and efficacy, setting the stage for a promising future in medical treatment.
Video synopsis is AI-generated and reviewed by AJMC® editorial staff.
