Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

RNAi emerged as a prospective molecular therapy nearly 15 years ago. Since then, two major RNAi platforms have been under development: oligonucleotides and gene therapy. Oligonucleotide-based approaches have seen more advancement, with some promising therapies that may soon reach market. In contrast...

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Bibliographic Details
Main Authors: Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne, Allison M. Fowler, Jocelyn O. Eidahl, Jacqueline S. Domire, Danielle A. Griffin, Adam C. Herman, Zarife Sahenk, Louise R. Rodino-Klapac, Scott Q. Harper
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene therapy
RNA interference
RNAi
adeno-associated viral vectors
AAV
facioscapulohumeral muscular dystrophy
FSHD
DUX4
microRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117301304

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