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

Biotherapies of neuromuscular disorders

J-F Briand  1 , M-O Roy  2 , F Mourlane  2 , C André  2 , N Loux  2 , C Rougeau  2 , T Toursel  2 , S Braun  2
Affiliations
Review

Biotherapies of neuromuscular disorders

J-F Briand et al. Rev Neurol (Paris). 2014 Dec.

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.

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