Article
Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin.
Scientific reports - 23 Jun 2020
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
Abstract excerpt
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 in the HBB cluster or related genes, compensate this disparity through γ-globin expression and subsequent fetal...
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