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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Vydáno v:Sci Rep
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2020
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Article
On-line přístup: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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