Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

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Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

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wetenschappelijk artikel

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наукова стаття, опублікована у вересні 2018

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Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

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Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

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Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

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Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

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Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

@en

Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

@nl

P1476

Disruption of the BCL11A Eryth ...... ients with β-Thalassemia Major

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Andreas Reik

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Dana N Levasseur

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Demetri Dalas

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Evangelia Yannaki

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Fyodor D Urnov

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Michael C Holmes

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Nikoletta Psatha

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Susan Phelps

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Thalia Papayannopoulou

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Yuanyue Zhou

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P2860

Human T gamma globin chain is a variant of A gamma chain (A gamma Sardinia)

Fast gapped-read alignment with Bowtie 2

Beta-thalassemia

Ex vivo generation of fully mature human red blood cells from hematopoietic stem cells

Loss of alpha-hemoglobin-stabilizing protein impairs erythropoiesis and exacerbates beta-thalassemia

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients

Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs

Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin.

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.

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313-326

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scholarly article

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10.1016/J.OMTM.2018.08.003

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English

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2018-09-21T00:00:00Z

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30182035

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6120587

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