Gene therapy for Pompe disease

Guest: Dr. Dwight Koeberl, Professor of Pediatrics, Professor of Molecular Genetics and Microbiology; Medical Director, Pediatric Biochemical Genetics Laboratory, Duke University
Topics: Gene therapy clinical trial for Pompe disease, gene therapy history and current practice, enzyme replacement therapy, newborn screening, Actus Therapeutics

 

Dr. Dwight Koeberl

 

Dwight’s faculty page

ClinicalTrials.gov listing for Pompe gene therapy trial

Dr. Kishnani’s faculty page

Actus Therapeutics

“Gene therapy trial for Pompe disease begins” DCRI press release, February 5, 2019

Click to Download: Radio In Vivo April 24, 2019

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CRISPR Gene Editing

Guest: Dr. Rodolphe Barrangou, Associate Professor, Food Science, NC State University
Topics: Gene editing system CRISPR, its history and the explosion of research in CRISPR in biotechnology, food and agriculture, translational medicine, disease modeling, gene therapy, and more

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Dr. Rodolphe Barrangou

 

NC State CRISPR Lab

Rodolphe’s faculty page, NCSU Department of Food, Bioprocessing,and Nutrition Sciences

“Steward of the Future: Rodolphe Barrangou”

CRISPR: Science Magazine’s Breakthrough of the Year, 2015

PubMed link to seminal 2007 paper, CRISPR provides acquired resistance against viruses in prokaryotes

Click to Download: Radio In Vivo May 4, 2016

Nobel Laureate Dr. Oliver Smithies

Guest: Dr. Oliver Smithies, Weatherspoon Eminent Distinguished Professor, Department of Pathology and Laboratory Medicine, UNC School of Medicine, winner of the 2007 Nobel Prize in Physiology or Medicine
Topics: Dr. Smithies’ distinguished career in science, his childhood, educational background, and outside interests; working with his wife, Dr. Nobuyu Maeda; gene targeting, gel electrophoresis, current work on pre-eclampsia

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Dr. Oliver Smithies

 

Dr. Smithies’ DPLM faculty profile

Dr. Maeda’s DPLM faculty profile

2007 UNC news release, “Oliver Smithies wins Nobel Prize”

Dr. Smithies’ Nobel lecture, December 7, 2007

Click to Download: Radio In Vivo October 28, 2015