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Keywords: IPSC models
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Journal Articles
Dis Model Mech (2017) 10 (6): 691–704.
Published: 1 June 2017
... 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 neuronal ceroid lipofuscinoses, Niemann-Pick types A and C1, and several...