Martin K. (Casey) Childers DO, PhD
University of Washington
Gene therapy for myotubular myopathy, a congenital muscle disease, that causes severe and fatal weakness of the breathing muscles is a focus of the Childers lab. Our international research group (led by Dr. Anna Buj-Bello, Genethon, Evry, France and Dr. Alan Beggs, Harvard, Boston) is working to develop this new gene therapy technology to translate findings in animals to treat human patients. The Childers lab also uses a technique to “reprogram” small samples of patient blood or urine into pluripotent stem cells that can be tested in a petri dish without harm to the patient. Using these powerful techniques our hope is to find and develop new therapy for inherited muscle diseases.
Dr. Childers's research article, Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy, was recently published in Science Translational Medicine. Click here for the full text and reprint of the article.
In more recent news, Audentes Therapeutics and Genethon have announced they have entered an agreement to develop treatment for X-Linked Myotubular Myopathy. A copy of the press release can be found here.
Dr. Childers played a role in founding Audentes and is currently a member of their Scientific Advisory Board. For more information regarding Audentes, please visit www.audentestx.com.