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

First Author: Hughes M

Attributed to: The development of gene therapy for Niemann-Pick type C 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

Parent Publication: Human Molecular Genetics

Issue: 17

ISSN: 0964-6906