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Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34(+) HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin
β-hemoglobinopathies are caused by abnormal or absent production of hemoglobin in the blood due to mutations in the β-globin gene (HBB). Imbalanced expression of adult hemoglobin (HbA) induces strong anemia in patients suffering from the disease. However, individuals with natural-occurring mutations...
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| I publikationen: | Sci Rep |
|---|---|
| Huvudupphovsmän: | , , , , , , , , , , , , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
Nature Publishing Group UK
2020
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7311455/ https://ncbi.nlm.nih.gov/pubmed/32576837 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-020-66309-x |
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