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Induced pluripotent stem cell models of lysosomal storage disorders

1 Jun 2017

Abstract excerpt

Induced pluripotent stem cells (iPSCs) have provided new opportunities to explore the cell biology and pathophysiology of human diseases, and the lysosomal storage disorder research community has been quick to adopt this technology. Patient-derived iPSC models have been generated for a number of lysosomal storage disorders, including Gaucher disease, Pompe disease, Fabry disease, metachromatic leukodystrophy, the...

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PMID: 28592657
DOI: 10.1242/dmm.029009

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