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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...
Autores principales: | Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Antony, Justin S., Epting, Thomas, Heumos, Lukas, Baskaran, Praveen, Admard, Jakob, Casadei, Nicolas, Latifi, Ngadhnjim, Siegmund, Darina M., Kormann, Michael S. D., Handgretinger, Rupert, Mezger, Markus |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7311455/ https://www.ncbi.nlm.nih.gov/pubmed/32576837 http://dx.doi.org/10.1038/s41598-020-66309-x |
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