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Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing
Disorders involving β-globin gene mutations, primarily β-thalassemia and sickle cell disease, represent a major target for hematopoietic stem/progenitor cell (HSPC) gene therapy. This includes CRISPR/Cas9-mediated genome editing approaches in adult CD34(+) cells aimed toward the reactivation of feta...
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| Yayımlandı: | Blood |
|---|---|
| Asıl Yazarlar: | , , , , , , , , , , |
| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
American Society of Hematology
2018
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| Konular: | |
| Online Erişim: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6024639/ https://ncbi.nlm.nih.gov/pubmed/29789357 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2018-03-838540 |
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