دورية أكاديمية
Oral mucosa-derived induced pluripotent stem cells from patients with ectrodactyly-ectodermal dysplasia-clefting syndrome
| العنوان: | Oral mucosa-derived induced pluripotent stem cells from patients with ectrodactyly-ectodermal dysplasia-clefting syndrome |
|---|---|
| المؤلفون: | Trevisan, Marta, Alvisi, Gualtiero, Barbaro, Vanessa, Barzon, Luisa, Raffa, Paolo, Migliorati, Angelo, Desole, Giovanna, Ruzittu, Silvia, Masi, Giulia, Di Iorio, Enzo, Palù, Giorgio |
| المساهمون: | Trevisan, Marta, Alvisi, Gualtiero, Barbaro, Vanessa, Barzon, Luisa, Raffa, Paolo, Migliorati, Angelo, Desole, Giovanna, Ruzittu, Silvia, Masi, Giulia, Di Iorio, Enzo, Palù, Giorgio |
| بيانات النشر: | Mary Ann Liebert Inc. |
| سنة النشر: | 2018 |
| المجموعة: | Padua Research Archive (IRIS - Università degli Studi di Padova) |
| مصطلحات موضوعية: | corneal epithelium, ectrodactyly-ectodermal dysplasia-clefting (EEC) syndrome, episomal vector, human induced pluripotent stem cells (hiPSCs), human oral mucosal epithelial stem cells (hOMESCs), reprogramming, Sendai virus vector, Biotechnology, Developmental Biology, Cell Biology |
| الوصف: | Ectrodactyly-Ectodermal dysplasia-Clefting (EEC) syndrome is a rare monogenic disease with autosomal dominant inheritance caused by mutations in the TP63 gene, leading to progressive corneal keratinocyte loss, limbal stem cell deficiency (LSCD), and eventually blindness. Currently, there is no treatment available to cure or slow down the keratinocyte loss. Human oral mucosal epithelial stem cells (hOMESCs), which are a mixed population of keratinocyte precursor stem cells, are used as source of autologous tissue for treatment of bilateral LSCD. However, hOMESCs from EEC patients have a reduced life span due to TP63 mutations and cannot be used for autologous transplantation. Human induced pluripotent stem cells (hiPSCs) represent a potentially unlimited source of autologous limbal stem cell for EEC patients and can be genetically modified by genome editing technologies to correct the disease ex vivo before transplantation. In this study, we describe for the first time the generation of integration-free EEC-hiPSCs from hOMESCs of EEC patients by Sendai virus vector and episomal vector-based reprogramming. The generated hiPSC clones expressed pluripotency markers and were successfully differentiated into derivatives of the three germ layers, as well as toward corneal epithelium. These cells may be used for EEC disease modeling and open perspectives for applications in cell therapy of LSCD. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | info:eu-repo/semantics/altIdentifier/pmid/29989433; info:eu-repo/semantics/altIdentifier/wos/WOS:000438172800001; volume:20; issue:4; firstpage:215; lastpage:224; numberofpages:10; journal:CELLULAR REPROGRAMMING; http://hdl.handle.net/11577/3279043Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85051245439; http://www.liebertonline.com/cellTest |
| DOI: | 10.1089/cell.2017.0064 |
| الإتاحة: | https://doi.org/10.1089/cell.2017.0064Test http://hdl.handle.net/11577/3279043Test http://www.liebertonline.com/cellTest |
| رقم الانضمام: | edsbas.7C961F90 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1089/cell.2017.0064 |
|---|