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Cell Stem Cell DOI:10.1016/j.stem.2015.06.013

From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial.

Publication TypeJournal Article
Year of Publication2015
AuthorsMcNeish, J, Gardner, JP, Wainger, BJ, Woolf, CJ, Eggan, K
JournalCell Stem Cell
Volume17
Issue1
Pages8-10
Date Published2015 Jul 02
ISSN1875-9777
KeywordsAmyotrophic Lateral Sclerosis, Animals, Carbamates, Clinical Trials as Topic, Drug Discovery, Humans, Induced Pluripotent Stem Cells, Mice, Models, Neurological, Motor Neurons, Mutation, Phenylenediamines, Superoxide Dismutase, Superoxide Dismutase-1, Translational Medical Research
Abstract

While iPSCs have created unprecedented opportunities for drug discovery, there remains uncertainty concerning the path to the clinic for candidate therapeutics discovered with their use. Here we share lessons that we learned, and believe are generalizable to similar efforts, while taking a discovery made using iPSCs into a clinical trial.

URLhttp://linkinghub.elsevier.com/retrieve/pii/S1934-5909(15)00273-8
DOI10.1016/j.stem.2015.06.013
Pubmed

http://www.ncbi.nlm.nih.gov/pubmed/26140603?dopt=Abstract

Alternate JournalCell Stem Cell
PubMed ID26140603
Grant ListR01 NS038253 / NS / NINDS NIH HHS / United States
/ / Howard Hughes Medical Institute / United States