Episode 10 - Gene Therapy

Looking to the future of therapeutics, the PhD 3 take a deep dive into gene replace therapy. Amr walks us through how scientists have developed novel means for the delivery of new functional genes into patients with rare diseases caused by mutations in in their DNA. As an recent example, we also explore the recently FDA approved gene therapy for a fatal disease known as spinal muscular atrophy (SMA). https://linktr.ee/notyetadr Edited by: Sienna Questions or Suggestions? Email us at [e-mail]