Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation.
Sign Up to like & getrecommendations! Published in 2018 at "Methods in molecular biology"
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
Functional muscle recovery following dystrophin and myostatin exon splice modulation in aged mdx mice.
Sign Up to like & getrecommendations! Published in 2019 at "Human molecular genetics"
DOI: 10.1093/hmg/ddz125
Abstract: Duchenne muscular dystrophy (DMD) is a rare genetic disease affecting 1 in 3500-5000 newborn boys. It is due to mutations in the DMD gene with a consequent lack of dystrophin protein that leads to deterioration… read more here.
Keywords: dmd; dystrophin myostatin; muscle; mdx mice ... See more keywords