Gene Therapy: RNA Therapeutics & Monogenic Diseases

This lecture covers gene therapy, RNA-based therapeutics, and the treatment of monogenic diseases. It discusses the strategies, challenges, and targets of gene therapy, as well as the development and approval of RNA therapeutics. The use of Nusinersen in spinal muscular atrophy and the advancements in severe combined immunodeficiency disease treatment through gene therapy are also explored. Additionally, the lecture delves into CAR T cell therapy for cancer treatment and the innovative CRISPR-based gene-editing therapy for hereditary blindness disorders.