title: RNA-targeting approaches for neuromuscular diseases.
abstract: Although most molecular therapy strategies for genetic diseases are based on gene replacement, interesting alternative approaches target RNA. These strategies rely on the modification of the mutated gene's expression in vivo by modulating pre-mRNA splicing, mRNA stability or mRNA translation. Here, we review recent progress using these RNA-based approaches in the field of muscle and muscle-related genetic diseases. Different molecular tools, including modified antisense oligonucleotides, pre-mRNA trans-splicing molecules, ribozymes or chemical compounds have been used successfully on patient cells or animal models of disease. These diverse strategies show tremendous therapeutic potential and several clinical trials have been initiated with Duchenne muscular dystrophy patients with promising results.
authors.affiliation: Généthon, CNRS/UEVE UMR8587 LAMBE, 1, rue de l'Internationale, 91000 Evry, France.
authors.forename: Florence
authors.initials: F
authors.lastname: Le Roy
authors.forename: Karine
authors.initials: K
authors.lastname: Charton
authors.forename: Christian L
authors.initials: CL
authors.lastname: Lorson
authors.forename: Isabelle
authors.initials: I
authors.lastname: Richard
mesh_terms: D005786:Gene Expression Regulation; D015316:Genetic Therapy; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics / Q000378:metabolism / Q000628:therapy; D009468:Neuromuscular Diseases / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D034622:RNA Interference; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism
chemical_list: D012333:RNA, Messenger