New treatment platform delivers full-length mRNA for Duchenne muscular dystrophy
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A new treatment platform developed by researchers at The University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, successfully restoring the production of an important muscle protein, dystrophin, and dramatically improving muscle strength, endurance and function in vivo.
 
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