دورية أكاديمية
Non-viral ex vivo genome-editing in mouse bona fide hematopoietic stem cells with CRISPR/Cas9
| العنوان: | Non-viral ex vivo genome-editing in mouse bona fide hematopoietic stem cells with CRISPR/Cas9 |
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| المؤلفون: | Suvd Byambaa, Hideki Uosaki, Tsukasa Ohmori, Hiromasa Hara, Hitoshi Endo, Osamu Nureki, Yutaka Hanazono |
| المصدر: | Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 451-462 (2021) |
| بيانات النشر: | Elsevier, 2021. |
| سنة النشر: | 2021 |
| المجموعة: | LCC:Genetics LCC:Cytology |
| مصطلحات موضوعية: | CRISPR/Cas9, non-viral genome editing, hematopoietic stem cells, homology-independent targeted integration, X-linked severe combined immunodeficiency, Genetics, QH426-470, Cytology, QH573-671 |
| الوصف: | We conducted two lines of genome-editing experiments of mouse hematopoietic stem cells (HSCs) with the clustered regularly interspaced short palindromic repeat (CRISPR) and CRISPR-associated protein 9 (Cas9). First, to evaluate the genome-editing efficiency in mouse bona fide HSCs, we knocked out integrin alpha 2b (Itga2b) with Cas9 ribonucleoprotein (Cas9/RNP) and performed serial transplantation in mice. The knockout efficiency was estimated at approximately 15%. Second, giving an example of X-linked severe combined immunodeficiency (X-SCID) as a target genetic disease, we showed a proof-of-concept of universal gene correction, allowing rescue of most of X-SCID mutations, in a completely non-viral setting. We inserted partial cDNA of interleukin-2 receptor gamma chain (Il2rg) into intron 1 of Il2rg via non-homologous end-joining (NHEJ) with Cas9/RNP and a homology-independent targeted integration (HITI)-based construct. Repaired HSCs reconstituted T lymphocytes and thymuses in SCID mice. Our results show that a non-viral genome-editing of HSCs with CRISPR/Cas9 will help cure genetic diseases. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 2329-0501 |
| Relation: | http://www.sciencedirect.com/science/article/pii/S2329050121000012; https://doaj.org/toc/2329-0501 |
| DOI: | 10.1016/j.omtm.2021.01.001 |
| URL الوصول: | https://doaj.org/article/2f08446944e44cac965321da03925c00 |
| رقم الأكسشن: | edsdoj.2f08446944e44cac965321da03925c00 |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 23290501 |
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| DOI: | 10.1016/j.omtm.2021.01.001 |