Screening chimeric GAA variants in preclinicalstudy results in hematopoietic stem cell genetherapy candidate vectors for Pompe disease

Screening chimeric GAA variants in preclinicalstudy results in hematopoietic stem cell genetherapy candidate vectors for Pompe disease

Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and cen...

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Bibliographic Details
Main Authors: Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Tim Maiwald, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hematopoietic stem and progenitor cells
lentiviral vector
Pompe disease
tag technology
glycosylation-independent lysosomal targeting
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122001590

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