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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 of the mucopolysaccharidoses. Here, we review the strategies employed for reprogramming and differentiation, as well as insights into disease etiology gleaned from the currently available models. Examples are provided to illustrate how iPSC-derived models can be employed to develop new therapeutic strategies for these disorders. We also discuss how models of these rare diseases could contribute to an enhanced understanding of more common neurodegenerative disorders such as Parkinson\u2019s disease, and discuss key challenges and opportunities in this area of research.<\/jats:p>","DOI":"10.1242\/dmm.029009","type":"journal-article","created":{"date-parts":[[2017,6,7]],"date-time":"2017-06-07T13:25:11Z","timestamp":1496841911000},"page":"691-704","source":"Crossref","is-referenced-by-count":28,"title":["Induced pluripotent stem cell models of lysosomal storage disorders"],"prefix":"10.1242","volume":"10","author":[{"given":"Daniel K.","family":"Borger","sequence":"first","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"McMahon","sequence":"additional","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tamanna","family":"Roshan Lal","sequence":"additional","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny","family":"Serra-Vinardell","sequence":"additional","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elma","family":"Aflaki","sequence":"additional","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3019-8500","authenticated-orcid":false,"given":"Ellen","family":"Sidransky","sequence":"additional","affiliation":[{"name":"Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"237","reference":[{"key":"2021042500140774800_DMM029009C1","doi-asserted-by":"publisher","first-page":"1276","DOI":"10.1038\/nbt.1503","article-title":"Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes","volume":"26","author":"Aasen","year":"2008","journal-title":"Nat. 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