<p><strong> </strong>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.</p><p><strong>Show Notes: </strong></p><p><strong>0:00 </strong>Intro to The Genetics Podcast</p><p><strong>00:59</strong> Welcome to Jeffrey</p><p><strong>01:33</strong> Early discovery of the dystrophin gene and how it shaped Duchenne muscular dystrophy (DMD) research</p><p><strong>09:07 </strong>Efforts to map dystrophin and develop practical diagnostic techniques</p><p><strong>12:04 </strong>How research in Jeffrey’s lab gradually led to the creation of micro-dystrophin </p><p><strong>20:15 </strong>How micro-dystrophin and AAV delivery converged into a viable systemic gene therapy strategy</p><p><strong>27:23</strong> Current successes and safety challenges in systemic AAV gene therapy for neuromuscular disease</p><p><strong>34:44 </strong>Prospects and limitations of gene editing for Duchenne and emerging alternatives to AAV micro-dystrophin</p><p><strong>44:57 </strong>Closing remarks</p><p>Please consider rating and reviewing us on your chosen podcast listening platform! </p><p><a href="https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link"><u>https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link</u></a></p><p><br></p>

The Genetics Podcast

Sano Genetics

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

DEC 4, 202547 MIN
The Genetics Podcast

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

DEC 4, 202547 MIN

Description

<p><strong> </strong>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.</p><p><strong>Show Notes: </strong></p><p><strong>0:00 </strong>Intro to The Genetics Podcast</p><p><strong>00:59</strong> Welcome to Jeffrey</p><p><strong>01:33</strong> Early discovery of the dystrophin gene and how it shaped Duchenne muscular dystrophy (DMD) research</p><p><strong>09:07 </strong>Efforts to map dystrophin and develop practical diagnostic techniques</p><p><strong>12:04 </strong>How research in Jeffrey’s lab gradually led to the creation of micro-dystrophin </p><p><strong>20:15 </strong>How micro-dystrophin and AAV delivery converged into a viable systemic gene therapy strategy</p><p><strong>27:23</strong> Current successes and safety challenges in systemic AAV gene therapy for neuromuscular disease</p><p><strong>34:44 </strong>Prospects and limitations of gene editing for Duchenne and emerging alternatives to AAV micro-dystrophin</p><p><strong>44:57 </strong>Closing remarks</p><p>Please consider rating and reviewing us on your chosen podcast listening platform! </p><p><a href="https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link"><u>https://drive.google.com/file/d/1Bp2_wVNSzntTs_zuoizU8bX1dvao4jfj/view?usp=share_link</u></a></p><p><br></p>