دورية أكاديمية
A Comprehensive, Ethnically Diverse Library of Sickle Cell Disease-Specific Induced Pluripotent Stem Cells
| العنوان: | A Comprehensive, Ethnically Diverse Library of Sickle Cell Disease-Specific Induced Pluripotent Stem Cells |
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| المؤلفون: | Seonmi Park, Andreia Gianotti-Sommer, Francisco Javier Molina-Estevez, Kim Vanuytsel, Nick Skvir, Amy Leung, Sarah S. Rozelle, Elmutaz Mohammed Shaikho, Isabelle Weir, Zhihua Jiang, Hong-Yuan Luo, David H.K. Chui, Maria Stella Figueiredo, Abdulraham Alsultan, Amein Al-Ali, Paola Sebastiani, Martin H. Steinberg, Gustavo Mostoslavsky, George J. Murphy |
| المصدر: | Stem Cell Reports, Vol 8, Iss 4, Pp 1076-1085 (2017) |
| بيانات النشر: | Elsevier, 2017. |
| سنة النشر: | 2017 |
| المجموعة: | LCC:Medicine (General) LCC:Biology (General) |
| مصطلحات موضوعية: | Medicine (General), R5-920, Biology (General), QH301-705.5 |
| الوصف: | Summary: Sickle cell anemia affects millions of people worldwide and is an emerging global health burden. As part of a large NIH-funded NextGen Consortium, we generated a diverse, comprehensive, and fully characterized library of sickle-cell-disease-specific induced pluripotent stem cells (iPSCs) from patients of different ethnicities, β-globin gene (HBB) haplotypes, and fetal hemoglobin (HbF) levels. iPSCs stand to revolutionize the way we study human development, model disease, and perhaps eventually, treat patients. Here, we describe this unique resource for the study of sickle cell disease, including novel haplotype-specific polymorphisms that affect disease severity, as well as for the development of patient-specific therapeutics for this phenotypically diverse disorder. As a complement to this library, and as proof of principle for future cell- and gene-based therapies, we also designed and employed CRISPR/Cas gene editing tools to correct the sickle hemoglobin (HbS) mutation. : In this resource article, Mostoslavsky, Murphy, and colleagues of the NextGen consortium describe a diverse, comprehensive, and characterized library of sickle cell disease-specific induced pluripotent stem cells (iPSCs) from patients of different ethnicities, β-globin gene (HBB) haplotypes and fetal hemoglobin (HbF) levels. This bank is readily available and accessible to all investigators. Keywords: induced pluripotent stem cells, iPSCs, sickle cell disease, disease modeling, directed differentiation, gene correction |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 2213-6711 |
| العلاقة: | http://www.sciencedirect.com/science/article/pii/S2213671116303071Test; https://doaj.org/toc/2213-6711Test |
| DOI: | 10.1016/j.stemcr.2016.12.017 |
| الوصول الحر: | https://doaj.org/article/52d22f01919b4214b5dcc432e367860cTest |
| رقم الانضمام: | edsdoj.52d22f01919b4214b5dcc432e367860c |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 22136711 |
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| DOI: | 10.1016/j.stemcr.2016.12.017 |