AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease-Reference-Cited by-同舟云学术

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

Published:2021-01 Issue:1 Volume:29 Page:162-175
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Johnson Tyler B.,White Katherine A.,Brudvig Jon J.,Cain Jacob T.,Langin Logan,Pratt Melissa A.,Booth Clarissa D.,Timm Derek J.,Davis Samantha S.,Meyerink Brandon,Likhite Shibi,Meyer Kathrin,Weimer Jill M.

Funder

Cure Batten CLN8 Velona Foundation

Amicus Therapeutics

Sanford Research Imaging Core

Sanford Research Center for Pediatric Research

NIH

Sanford Research Molecular Pathology Core

Sanford Research Center for Cancer Biology

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference47 articles.

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4. Mitochondrial ATP synthase subunit c storage in the ceroid-lipofuscinoses (Batten disease);Palmer;Am. J. Med. Genet.,1992

5. CLN8 is an endoplasmic reticulum cargo receptor that regulates lysosome biogenesis;di Ronza;Nat. Cell Biol.,2018

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