Supercharged Oligonucleotides Show Promise for Duchenne Muscular Dystrophy

Supercharged Oligonucleotides Show Promise for Duchenne Muscular Dystrophy

A supercharged oligonucleotide therapeutic has shown potential for maximizing the benefits of a genetic treatment for Duchenne muscular dystrophy (DMD) in preclinical studies. The study, in PNAS, establishes the value of these bifunctional or bipartite antisense oligonucleotides (ASOs)—short, synthetic, single-stranded nucleic acid analogs comprising an antisense portion with a regulatory tail that recruits splicing suppressors—in RNA-targeted t…

Seven biotechs driving progress in Duchenne muscular dystrophy

In this article, we take a look at seven clinical-stage biotechs that are fostering R&D in the Duchenne muscular dystrophy therapeutic space. The post Seven biotechs driving progress in Duchenne muscular dystrophy  appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and lin…