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Concise Review: Human Induced Pluripotent Stem Cell Models of Retinitis Pigmentosa
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SYSNO ASEP 0493037 Druh ASEP J - Článek v odborném periodiku Zařazení RIV J - Článek v odborném periodiku Poddruh J Článek ve WOS Název Concise Review: Human Induced Pluripotent Stem Cell Models of Retinitis Pigmentosa Tvůrce(i) Castro, A.A. (ES)
Lukovic, D. (ES)
Jendelová, Pavla (UEM-P) RID, ORCID
Erceg, Slaven (UEM-P) RID, ORCIDZdroj.dok. Stem Cells. - : Oxford University Press - ISSN 1066-5099
Roč. 36, č. 4 (2018), s. 474-481Poč.str. 8 s. Jazyk dok. eng - angličtina Země vyd. US - Spojené státy americké Klíč. slova differentiation ; gene targeting ; induced pluripotent stem cells ; induced pluripotent stem Vědní obor RIV FH - Neurologie, neurochirurgie, neurovědy Obor OECD Cell biology CEP EF15_003/0000419 GA MŠMT - Ministerstvo školství, mládeže a tělovýchovy GBP304/12/G069 GA ČR - Grantová agentura ČR Institucionální podpora UEM-P - RVO:68378041 UT WOS 000428702800002 EID SCOPUS 85041662200 DOI 10.1002/stem.2783 Anotace Hereditary retinal dystrophies, specifically retinitis pigmentosa (RP) are clinically and genetically heterogeneous diseases affecting primarily retinal cells and retinal pigment epithelial cells with blindness as a final outcome. Understanding the pathogenicity behind these diseases has been largely precluded by the unavailability of affected tissue from patients, large genetic heterogeneity and animal models that do not faithfully represent some human diseases. A landmark discovery of human induced pluripotent stem cells (hiPSCs) permitted the derivation of patient-specific cells. These cells have unlimited self-renewing capacity and the ability to differentiate into RP-affected cell types, allowing the studies of disease mechanism, drug discovery, and cell replacement therapies, both as individual cell types and organoid cultures. Together with precise genome editing, the patient specific hiPSC technology offers novel strategies for targeting the pathogenic mutations and design therapies toward retinal dystrophies. This study summarizes current hiPSC-based RP models and highlights key achievements and challenges of these cellular models, as well as questions that still remain unanswered. Pracoviště Ústav experimentální medicíny Kontakt Lenka Koželská, lenka.kozelska@iem.cas.cz, Tel.: 241 062 218, 296 442 218 Rok sběru 2019
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