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† Department of Chemistry, School of Arts and Sciences, The Johns Hopkins University, 3400 North Charles Street, Baltimore, Maryland 21218, United States ‡ W. Harry Feinstone Department of Molecular ...
Satellos Bioscience Chief Scientific Officer discusses early-stage trials of the company's Duchenne muscular dystrophy drug, ...
In many genetic diseases, a small error in the DNA code can have devastating effects. These errors, called nonsense mutations ...
University of Illinois at Chicago, 833 South Wood Street, Chicago, Illinois 60612 Article Views are the COUNTER-compliant sum of full text article downloads since November 2008 (both PDF and HTML) ...
Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) technology to deliver a gene designed to produce a shortened form of the ...