Generation of two induced pluripotent stem cell lines (CHOCi002-A and CHOCi003-A) from Pompe disease patients with compound heterozygous mutations in the GAA gene

Generation of two induced pluripotent stem cell lines (CHOCi002-A and CHOCi003-A) from Pompe disease patients with compound heterozygous mutations in the GAA gene

Pompe disease is an autosomal recessive lysosomal storage disease caused by pathogenic variants in GAA, which encodes an enzyme integral to glycogen catabolism, acid α-glucosidase. Disease-relevant cell lines are necessary to evaluate the efficacy of genotype-specific therapies. Dermal fibroblasts f...

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Bibliographic Details
Main Authors: Chloe Christensen, Perla Heckman, Allisandra Rha, Shih-Hsin Kan, Jerry Harb, Raymond Wang
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Stem Cell Research
Subjects:
Pompe disease
Lysosomal storage disorder
Induced pluripotent stem cells
Sendai virus
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506123001034

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http://www.sciencedirect.com/science/article/pii/S1873506123001034

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