Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.
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DOI: 10.1007/978-1-4939-8651-4_21
Abstract: Using antisense oligonucleotides (AOs) to reframe mutated dystrophin, a recently developed therapeutic approach for Duchenne muscular dystrophy (DMD) named exon skipping, is considered among the most promising treatments for DMD patients. The development of this… read more here.
Keywords: myostatin; dystrophin myostatin; muscle; administration antisense ... See more keywords