Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
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Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.Emerging cellular and gene therapies for congenital anemias.Gene Therapy for β-Hemoglobinopathies.Strict in vivo specificity of the Bcl11a erythroid enhancer.Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies.A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies.Concise Review: Epigenetic Regulation of Hematopoiesis: Biological Insights and Therapeutic Applications.Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.Emerging Therapies.Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.DROSHA Knockout Leads to Enhancement of Viral Titers for Vectors Encoding miRNA-Adapted shRNAsTRIAMF: A New Method for Delivery of Cas9 Ribonucleoprotein Complex to Human Hematopoietic Stem CellsDisruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia MajorGene Therapy for Hemoglobinopathies
P2860
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P2860
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 06 September 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@en
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@nl
type
label
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@en
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@nl
prefLabel
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@en
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@nl
P2093
P2860
P356
P1476
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.
@en
P2093
Christian Brendel
Daniel E Bauer
David A Williams
Denise Klatt
Jonathan Fogel
Matthew C Canver
Matthew M Heeney
Richard I Gregory
Stuart H Orkin
Swaroopa Guda
P2860
P304
P356
10.1172/JCI87885
P407
P577
2016-09-06T00:00:00Z