AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease. (2018)

First Author: Hughes MP

Attributed to: Gene therapy for CLN5 Batten disease funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.1093/hmg/ddy212

PubMed Identifier: 29878115

Publication URI: http://europepmc.org/abstract/MED/29878115

Type: Journal Article/Review

Volume: 27

Parent Publication: Human molecular genetics

Issue: 17

ISSN: 0964-6906