title: Biotherapies of neuromuscular disorders.
abstract: This review focuses on the most recent data on biotherapeutic approaches, using DNA, RNA, recombinant proteins, or cells as therapeutic tools or targets for the treatment of neuromuscular diseases. Many of these novel technologies have now reached the clinical stage and have or are about to move to the market. Others, like genome editing are still in an early stage but hold great promise.
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France. Electronic address: jfbriand@afm-telethon.fr.
authors.forename: J-F
authors.initials: JF
authors.lastname: Briand
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: M-O
authors.initials: MO
authors.lastname: Roy
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: F
authors.initials: F
authors.lastname: Mourlane
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: C
authors.initials: C
authors.lastname: André
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: N
authors.initials: N
authors.lastname: Loux
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: C
authors.initials: C
authors.lastname: Rougeau
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: T
authors.initials: T
authors.lastname: Toursel
authors.affiliation: AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
authors.forename: S
authors.initials: S
authors.lastname: Braun
mesh_terms: D000818:Animals; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D001327:Autoimmune Diseases / Q000628:therapy; D001691:Biological Therapy / Q000379:methods*; D015316:Genetic Therapy; D006801:Humans; D009046:Motor Neurons / Q000378:metabolism; D018482:Muscle, Skeletal / Q000378:metabolism; D009468:Neuromuscular Diseases / Q000628:therapy*; D012313:RNA / Q000627:therapeutic use; D017393:RNA Editing / Q000235:genetics
chemical_list: D000911:Antibodies, Monoclonal; D012313:RNA
keywords: Anticorps monoclonaux; Biotherapies; Biothérapies; Gene therapy; Modulation de l’ARN; Monoclonal antibodies; Motoneuron; Motoneurone; Muscle; RNA modulation; Thérapie génique