Gene Therapy for ALS—A Perspective

Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure. Recent advances in gene therapy open a new perspective to treat this disorder—particularly for the characterized genetic forms. Gene therapy approaches, involving the delivery of antisense oligonucleoti...

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Bibliographic Details
Main Authors: Marisa Cappella, Chiara Ciotti, Mathilde Cohen-Tannoudji, Maria Grazia Biferi
Format: Article
Language:English
Published: MDPI AG 2019-09-01
Series:International Journal of Molecular Sciences
Subjects:
AAV
Online Access:https://www.mdpi.com/1422-0067/20/18/4388