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Episode 10 - Gene Therapy
Manage episode 332168933 series 3353692
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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).
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Edited by: Sienna
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