Počet záznamů: 1
hiPSC Disease Modeling of Rare Hereditary Cerebellar Ataxias: Opportunities and Future Challenges
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SYSNO ASEP 0478710 Druh ASEP J - Článek v odborném periodiku Zařazení RIV J - Článek v odborném periodiku Poddruh J Článek ve WOS Název hiPSC Disease Modeling of Rare Hereditary Cerebellar Ataxias: Opportunities and Future Challenges Tvůrce(i) Lukovic, D. (ES)
Moreno-Manzano, V. (ES)
Rodriquez - Jimenez, F.J. (ES)
Vilches, A. (ES)
Syková, Eva (UEM-P) RID
Jendelová, Pavla (UEM-P) RID, ORCID
Stojkovic, M. (ES)
Erceg, Slaven (UEM-P) RID, ORCIDZdroj.dok. Neuroscientist - ISSN 1073-8584
Roč. 23, č. 5 (2017), s. 554-566Poč.str. 13 s. Jazyk dok. eng - angličtina Země vyd. US - Spojené státy americké Klíč. slova 3D organoids ; ataxia ; disease modelling Vědní obor RIV EB - Genetika a molekulární biologie Obor OECD Developmental biology CEP GBP304/12/G069 GA ČR - Grantová agentura ČR LO1309 GA MŠMT - Ministerstvo školství, mládeže a tělovýchovy ED1.1.00/02.0109 GA MŠMT - Ministerstvo školství, mládeže a tělovýchovy Institucionální podpora UEM-P - RVO:68378041 UT WOS 000412270000014 EID SCOPUS 85029509414 DOI 10.1177/1073858416672652 Anotace Cerebellar ataxias are clinically and genetically heterogeneous diseases affecting primary cerebellar cells. The lack of availability of affected tissue from cerebellar ataxias patients is the main obstacle in investigating the pathogenicity of these diseases. The landmark discovery of human-induced pluripotent stem cells (hiPSC) has permitted the derivation of patient-specific cells with an unlimited self-renewing capacity. Additionally, their potential to differentiate into virtually any cell type of the human organism allows for large amounts of affected cells to be generated in culture, converting this hiPSC technology into a revolutionary tool in the study of the mechanisms of disease, drug discovery, and gene correction. In this review, we will summarize the current studies in which hiPSC were utilized to study cerebellar ataxias. Describing the currently available 2D and 3D hiPSC-based cellular models, and due to the fact that extracerebellar cells were used to model these diseases, we will discuss whether or not they represent a faithful cellular model and whether they have contributed to a better understanding of disease mechanisms. Pracoviště Ústav experimentální medicíny Kontakt Lenka Koželská, lenka.kozelska@iem.cas.cz, Tel.: 241 062 218, 296 442 218 Rok sběru 2018
Počet záznamů: 1