Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease
Author:
Funder
Canterbury Medical Research Foundation
Batten Disease Support and Research Association
Publisher
Elsevier BV
Subject
Cellular and Molecular Neuroscience,Sensory Systems,Ophthalmology
Reference57 articles.
1. Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs;Boyd;Gene Ther.,2016
2. Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs;Boyd;Gene Ther.,2016
3. Brms: an R package for bayesian multilevel models using stan;Bürkner;J. Stat. Software,2017
4. Towards a new understanding of NCL pathogenesis;Cooper;BBA-Mol. Basis. Dis.,2015
5. In vivo–directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous;Dalkara;Sci. Transl. Med.,2013
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