{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T10:47:14Z","timestamp":1792666034721,"version":"4.5.0"},"reference-count":73,"publisher":"Oxford University Press (OUP)","issue":"7","license":[{"start":{"date-parts":[[2017,5,22]],"date-time":"2017-05-22T00:00:00Z","timestamp":1495411200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2017,7,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>High attrition rates and loss of capital plague the drug discovery process. This is particularly evident for mitochondrial disease that typically involves neurological manifestations and is caused by nuclear or mitochondrial DNA defects. This group of heterogeneous disorders is difficult to target because of the variability of the symptoms among individual patients and the lack of viable modeling systems. The use of induced pluripotent stem cells (iPSCs) might significantly improve the search for effective therapies for mitochondrial disease. iPSCs can be used to generate patient-specific neural cell models in which innovative compounds can be identified or validated. Here we discuss the promises and challenges of iPSC-based drug discovery for mitochondrial disease with a specific focus on neurological conditions. We anticipate that a proper use of the potent iPSC technology will provide critical support for the development of innovative therapies against these untreatable and detrimental disorders.<\/jats:p>","DOI":"10.1002\/stem.2637","type":"journal-article","created":{"date-parts":[[2017,5,23]],"date-time":"2017-05-23T23:05:40Z","timestamp":1495580740000},"page":"1655-1662","source":"Crossref","is-referenced-by-count":37,"title":["Concise Review: Induced Pluripotent Stem Cell-Based Drug Discovery for Mitochondrial Disease"],"prefix":"10.1093","volume":"35","author":[{"given":"Gizem","family":"Inak","sequence":"first","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carmen","family":"Lorenz","sequence":"additional","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"},{"name":"b Berlin Institute of Health (BIH), Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pawel","family":"Lisowski","sequence":"additional","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"},{"name":"c Institute of Genetics and Animal Breeding, Department of Molecular Biology, Polish Academy of Sciences, Jastrzebiec, Magdalenka, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annika","family":"Zink","sequence":"additional","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"},{"name":"d Charit\u00e9 \u2013 Universit\u00e4tsmedizin, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Mlody","sequence":"additional","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9457-1952","authenticated-orcid":false,"given":"Alessandro","family":"Prigione","sequence":"additional","affiliation":[{"name":"a Max Delbrueck Center for Molecular Medicine (MDC), Mitochondrial and Cell Fate Reprogramming, Department of Neuroproteomics, Berlin, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2017,5,22]]},"reference":[{"key":"2022022509302223600_stem2637-bib-0001","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1038\/nrd3078","article-title":"How to improve R&D productivity: The pharmaceutical industry\u2019s grand 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