Human Gene Therapy, volume 36, issue 5-6, pages 637-652

AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration

Maria Carla Carisi 1
, 
Claire Shamber 1
, 
Martha Bishop 1
, 
Madison Sangster 1
, 
Uma Chandrachud 1
, 
Brandon Meyerink 2
, 
Louis Jean Pilaz 2
, 
Yulia Grishchuk 1
1
 
Center for Genomic Medicine and Department of Neurology, Massachusetts General Hospital Research Institute and Harvard Medical School, Boston, Massachusetts, USA
2
 
Pediatrics and Rare Diseases Group, Sanford Research Institute, University of South Dakota, Sioux Falls, South Dakota, USA.
Publication type: Journal Article
Publication date: 2025-03-01
scimago Q2
SJR: 1.091
CiteScore: 6.5
Impact factor: 3.9
ISSN: 10430342, 15577422
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