Targeted exon skipping of a CEP290 mutation rescues Joubert syndrome phenotypes in vitro and in a murine model. (2018)

First Author: Ramsbottom SA
Attributed to:  Models, modifiers and novel treatments of Joubert syndrome funded by MRC

Abstract

No abstract provided

Bibliographic Information

Digital Object Identifier: http://dx.doi.org/10.1073/pnas.1809432115

PubMed Identifier: 30446612

Publication URI: http://europepmc.org/abstract/MED/30446612

Type: Journal Article/Review

Volume: 115

Parent Publication: Proceedings of the National Academy of Sciences of the United States of America

Issue: 49

ISSN: 0027-8424