EP 216: Four decades advancing Duchenne research with Jeffrey Chamberlain, University of Washington скачать в хорошем качестве

EP 216: Four decades advancing Duchenne research with Jeffrey Chamberlain, University of Washington

Summary: This week on The Genetics Podcast, Patrick is joined by Dr. Jeffrey Chamberlain, Professor at the University of Washington School of Medicine, co-founder of Kinea Bio, and Director of the Muscular Dystrophy Cooperative Research Center of Seattle. They discuss the early breakthroughs that revealed the structure and function of the dystrophin gene, how those insights led to the creation of micro-dystrophin and systemic AAV delivery in Duchenne, and the major scientific and clinical challenges the field must now solve. Show Notes: 0:00 Intro to The Genetics Podcast 01:00 Welcome to Jeffrey 01:37 Early discovery of the dystrophin gene and how it shaped Duchenne muscular dystrophy (DMD) research 10:01 Efforts to map dystrophin and develop practical diagnostic techniques 13:14 How research in Jeffrey’s lab gradually led to the creation of micro-dystrophin 22:00 How micro-dystrophin and AAV delivery converged into a viable systemic gene therapy strategy 29:37 Current successes and safety challenges in systemic AAV gene therapy for neuromuscular disease 37:38 Prospects and limitations of gene editing for Duchenne and emerging alternatives to AAV micro-dystrophin 48:50 Closing remarks Please consider rating and reviewing us on your chosen podcast listening platform! https://drive.google.com/file/d/1Bp2_...