Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

Pompe disease is a lysosomal storage disorder caused by malfunctions of the acid alpha-glucosidase (GAA) enzyme with a consequent toxic accumulation of glycogen in cells. Muscle wasting and hypertrophic cardiomyopathy are the most common clinical signs that can lead to cardiac and respiratory failur...

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Main Authors: Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, Derek Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytan, Diego Leon-Rico, Neelam Panchal, Kathryn Whitmore, Miguel Calero, Sara Benedetti, Giorgia Santilli, Adrian J. Thrasher, H. Bobby Gaspar
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Pompe disease
hematopoietic stem cell gene therapy
lentiviral vector
acid alpha-glucosidase
GAA
erythroid-specific enhancer
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301509

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http://www.sciencedirect.com/science/article/pii/S2329050120301509

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