@article{ae795c7f4b08434198d22efca975b505,
title = "Morphological and Molecular Defects in Human Three-Dimensional Retinal Organoid Model of X-Linked Juvenile Retinoschisis",
abstract = "Chiou, Schlaeger, and colleagues use hiPSC-derived retinal organoids to model X-linked juvenile retinoschisis. They show that patient hiPSC-derived retinal organoids replicate key pathologies observed in patients, including retinal splitting and photoreceptor deficit. The observed abnormalities were normalized in organoids derived from isogenic CRISPR/Cas9 gene-corrected hiPSCs. This validated XLRS in vitro model could be used to test and optimize therapeutic approaches.",
keywords = "CRISPR/Cas9 gene editing, induced pluripotent stem cells, retinal degeneration, retinal organoid, retinogenesis, retinoschisin, RS1, X-linked juvenile retinoschisis",
author = "Huang, {Kang Chieh} and Wang, {Mong Lien} and Chen, {Shih Jen} and Kuo, {Jean Cheng} and Wang, {Won Jing} and {Nhi Nguyen}, {Phan Nguyen} and Wahlin, {Karl J.} and Lu, {Jyh Feng} and Tran, {Audrey A.} and Michael Shi and Yueh Chien and Yarmishyn, {Aliaksandr A.} and Tsai, {Ping Hsing} and Yang, {Tien Chun} and Jane, {Wann Neng} and Chang, {Chia Ching} and Peng, {Chi Hsien} and Schlaeger, {Thorsten M.} and Chiou, {Shih Hwa}",
note = "Funding Information: We thank Li-Ju Lin BS for excellent technical assistance. This study was funded by the Ministry of Science and Technology (MOST) of Taiwna and Academic Sinica of Taiwan ( 107-2633-B-009-003 ), Taipei Veterans General Hospital of Taiwan ( V107E-002-2 , V108E-006-4 , and V108D46-004-MY2-1 ), the Department of Health Cancer Center Research of Excellence of Taiwan ( MOHW106-TDU-B-211-144003 ), the Excellent Clinical Trail Center of Taiwan ( 107-TDU-B-211-123001/MOHW108-TDU-B-211-133001 ), NRPB Human iPSC Alliance-Core Service of Taiwan ( MOST 105-2325-B-010-005 ), VGH , TSGH , NDMC , AS Joint Research Program of Taiwan ( VTA105-V1-5-1/107-V1-5-1/V108-V1-5-1 ), VGH , NTUH Joint Research Program of Taiwan ( VN106-02/VN107-16/VN108-2 ), NHRI ( NHRI-EX107-10621BI ), and the “Center for Intelligent Drug Systems and Smart Bio-devices (IDS2B)” of NCTU , “Cancer Progression Research Center” of NYMU from The Featured Areas Research Center Program within the framework of the Higher Education Sprout Project by the Ministry of Education (MOE) in Taiwan. Publisher Copyright: {\textcopyright} 2019 The Authors",
year = "2019",
month = nov,
day = "12",
doi = "10.1016/j.stemcr.2019.09.010",
language = "English",
volume = "13",
pages = "906--923",
journal = "Stem Cell Reports",
issn = "2213-6711",
publisher = "Cell Press",
number = "5",
}