Intravitreal gene therapy preserves retinal function in a canine model of CLN2 neuronal ceroid lipofuscinosis-Reference-Cited by-同舟云学术

Intravitreal gene therapy preserves retinal function in a canine model of CLN2 neuronal ceroid lipofuscinosis

Published:2023-01 Issue: Volume:226 Page:109344
ISSN:0014-4835
Container-title:Experimental Eye Research
language:en
Short-container-title:Experimental Eye Research

Author:

Kick Grace Robinson,Whiting Rebecca E.H.,Ota-Kuroki Juri,Castaner Leilani J.,Morgan-Jack Brandie,Sabol Julianna C.,Meiman Elizabeth J.,Ortiz Francheska,Katz Martin L.^ORCID

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Cellular and Molecular Neuroscience,Sensory Systems,Ophthalmology

Reference50 articles.

1. A frame shift mutation in canine TPP1 (the ortholog of human CLN2) in a juvenile Dachshund with neuronal ceroid lipofuscinosis;Awano;Mol. Genet. Metabol.,2006

2. Long-term effect of gene therapy on leber's congenital amaurosis;Bainbridge;N. Engl. J. Med.,2015

3. Sorting of lysosomal proteins;Braulke;Biochim. Biophys. Acta Mol. Cell Res.,2009

4. CLN2;Chang,2011

5. Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: results from a Phase 1/2 study;Cherukuri;Clin. Immunol.,2018

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