@article{borger_induced_2017,
	title = {Induced pluripotent stem cell models of lysosomal storage disorders},
	volume = {10},
	issn = {1754-8403},
	url = {http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5483008/},
	doi = {10.1242/dmm.029009},
	abstract = {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 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’s disease, and discuss key challenges and opportunities in this area of research., Summary: This Review discusses how induced pluripotent stem cells (iPSCs) provide new opportunities to explore the biology and pathophysiology of lysosomal storage diseases, and how iPSCs have illuminated the role of lysosomes in more common disorders.},
	number = {6},
	urldate = {2017-07-28},
	journal = {Disease Models \& Mechanisms},
	author = {Borger, Daniel K. and McMahon, Benjamin and Roshan Lal, Tamanna and Serra-Vinardell, Jenny and Aflaki, Elma and Sidransky, Ellen},
	month = jun,
	year = {2017},
	pmid = {28592657},
	pmcid = {PMC5483008},
	pages = {691--704}
}

{"_id":"tbtKa6zTGsun9XRjG","bibbaseid":"borger-mcmahon-roshanlal-serravinardell-aflaki-sidransky-inducedpluripotentstemcellmodelsoflysosomalstoragedisorders-2017","authorIDs":[],"author_short":["Borger, D. K.","McMahon, B.","Roshan Lal, T.","Serra-Vinardell, J.","Aflaki, E.","Sidransky, E."],"bibdata":{"bibtype":"article","type":"article","title":"Induced pluripotent stem cell models of lysosomal storage disorders","volume":"10","issn":"1754-8403","url":"http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5483008/","doi":"10.1242/dmm.029009","abstract":"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 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’s disease, and discuss key challenges and opportunities in this area of research., Summary: This Review discusses how induced pluripotent stem cells (iPSCs) provide new opportunities to explore the biology and pathophysiology of lysosomal storage diseases, and how iPSCs have illuminated the role of lysosomes in more common disorders.","number":"6","urldate":"2017-07-28","journal":"Disease Models & Mechanisms","author":[{"propositions":[],"lastnames":["Borger"],"firstnames":["Daniel","K."],"suffixes":[]},{"propositions":[],"lastnames":["McMahon"],"firstnames":["Benjamin"],"suffixes":[]},{"propositions":[],"lastnames":["Roshan","Lal"],"firstnames":["Tamanna"],"suffixes":[]},{"propositions":[],"lastnames":["Serra-Vinardell"],"firstnames":["Jenny"],"suffixes":[]},{"propositions":[],"lastnames":["Aflaki"],"firstnames":["Elma"],"suffixes":[]},{"propositions":[],"lastnames":["Sidransky"],"firstnames":["Ellen"],"suffixes":[]}],"month":"June","year":"2017","pmid":"28592657","pmcid":"PMC5483008","pages":"691–704","bibtex":"@article{borger_induced_2017,\n\ttitle = {Induced pluripotent stem cell models of lysosomal storage disorders},\n\tvolume = {10},\n\tissn = {1754-8403},\n\turl = {http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5483008/},\n\tdoi = {10.1242/dmm.029009},\n\tabstract = {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 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’s disease, and discuss key challenges and opportunities in this area of research., Summary: This Review discusses how induced pluripotent stem cells (iPSCs) provide new opportunities to explore the biology and pathophysiology of lysosomal storage diseases, and how iPSCs have illuminated the role of lysosomes in more common disorders.},\n\tnumber = {6},\n\turldate = {2017-07-28},\n\tjournal = {Disease Models \\& Mechanisms},\n\tauthor = {Borger, Daniel K. and McMahon, Benjamin and Roshan Lal, Tamanna and Serra-Vinardell, Jenny and Aflaki, Elma and Sidransky, Ellen},\n\tmonth = jun,\n\tyear = {2017},\n\tpmid = {28592657},\n\tpmcid = {PMC5483008},\n\tpages = {691--704}\n}\n\n","author_short":["Borger, D. K.","McMahon, B.","Roshan Lal, T.","Serra-Vinardell, J.","Aflaki, E.","Sidransky, E."],"key":"borger_induced_2017","id":"borger_induced_2017","bibbaseid":"borger-mcmahon-roshanlal-serravinardell-aflaki-sidransky-inducedpluripotentstemcellmodelsoflysosomalstoragedisorders-2017","role":"author","urls":{"Paper":"http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5483008/"},"downloads":0,"html":""},"bibtype":"article","biburl":"https://bibbase.org/zotero/muneebsalie","creationDate":"2020-07-02T10:44:34.939Z","downloads":0,"keywords":[],"search_terms":["induced","pluripotent","stem","cell","models","lysosomal","storage","disorders","borger","mcmahon","roshan lal","serra-vinardell","aflaki","sidransky"],"title":"Induced pluripotent stem cell models of lysosomal storage disorders","year":2017,"dataSources":["QogkwkhcTZyMJJoTw"]}