| Autor: |
Mishra, Bibhu, Chou, Song, Lin, Michelle I, Paik, Elizabeth J, Zhang, Ying, Liang, Ruibin, Tomkinson, Kaleigh, Pettiglio, Michael, Sanginario, Andrew, Allen, Melanie, Cradick, Thomas, John, Matthias, Chakraborty, Tirtha, Cowan, Chad A, Novak, Rodger, Lundberg, Ante S |
| Zdroj: |
Blood; December 2016, Vol. 128 Issue: 22 p3623-3623, 1p |
| Abstrakt: |
Fetal hemoglobin (HbF) is abundant in the late stage fetus and newborns, but is progressively lost over the first 6 months of life as the genes encoding the g-globin subunit of HbF are repressed. HbF expression can provide clinical benefit to patients with deficient or defective b-globin, for example in β-thalassemia and Sickle Cell Disease (SCD), respectively. CRISPR-Cas9 technology offers a unique treatment modality that can be used to ex vivoedit regulatory DNA sequences in patient CD34+hematopoietic stem and progenitor cells (HSPC) containing hematopoietic stem and progenitor cells in order to upregulate HbF. Reinfusion of edited CD34+HSPC would be expected to lead to the production of erythrocytes expressing high levels of HbF, and amelioration of disease. |
| Databáze: |
Supplemental Index |
| Externí odkaz: |
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