Related Experiment Video
Updated: Apr 20, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Biotherapies of neuromuscular disorders
J-F Briand1, M-O Roy1, F Mourlane1
1AFM-Telethon, 1, rue de l'Internationale, BP 59, 91002 Evry cedex, France.
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.
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