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Induced pluripotent stem cells derived from mouse models of lysosomal storage disorders

12 Apr 2010

Abstract excerpt

Most lysosomal storage diseases (LSDs) are life-threatening genetic diseases. The pathogenesis of these diseases is poorly understood. Induced pluripotent stem (iPS) cell technology offers new opportunities for both mechanistic studies and development of stem cell- based therapies. Here we report the generation of disease-specific iPS cells from mouse models of Fabry disease, globoid cell leukodystrophy (GLD),...

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PMID: 20385825
DOI: 10.1073/pnas.1002758107

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