Biotherapies of neuromuscular disorders

J-F Briand; M-O Roy; F Mourlane; C André; N Loux; C Rougeau; T Toursel; S Braun

doi:10.1016/j.neurol.2014.07.018

Biotherapies of neuromuscular disorders

Rev Neurol (Paris). 2014 Dec;170(12):799-807. doi: 10.1016/j.neurol.2014.07.018. Epub 2014 Nov 13.

Authors

J-F Briand¹, M-O Roy², F Mourlane², C André², N Loux², C Rougeau², T Toursel², S Braun²

Affiliations

¹ AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France. Electronic address: jfbriand@afm-telethon.fr.
² AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.

PMID: 25459122
DOI: 10.1016/j.neurol.2014.07.018

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.

Keywords: Anticorps monoclonaux; Biotherapies; Biothérapies; Gene therapy; Modulation de l’ARN; Monoclonal antibodies; Motoneuron; Motoneurone; Muscle; RNA modulation; Thérapie génique.

Publication types

Review

MeSH terms

Animals
Antibodies, Monoclonal / therapeutic use
Autoimmune Diseases / therapy
Biological Therapy / methods*
Genetic Therapy
Humans
Motor Neurons / metabolism
Muscle, Skeletal / metabolism
Neuromuscular Diseases / therapy*
RNA / therapeutic use
RNA Editing / genetics

Substances

Antibodies, Monoclonal
RNA