Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin.
about
Customizing the genome as therapy for the β-hemoglobinopathiesThe therapeutic potential of genome editing for β-thalassemiaEnabling functional genomics with genome engineeringSystematic Functional Dissection of Common Genetic Variation Affecting Red Blood Cell TraitsAssociation between Variants at BCL11A Erythroid-Specific Enhancer and Fetal Hemoglobin Levels among Sickle Cell Disease Patients in Cameroon: Implications for Future Therapeutic InterventionsGenetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Insight into GATA1 transcriptional activity through interrogation of cis elements disrupted in human erythroid disordersOriginal Research: Generation of non-deletional hereditary persistence of fetal hemoglobin β-globin locus yeast artificial chromosome transgenic mouse models: -175 Black HPFH and -195 Brazilian HPFHTranscription factor-DNA binding: beyond binding site motifs.Gene Therapy for β-Hemoglobinopathies.Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies.A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.Concise Review: Epigenetic Regulation of Hematopoiesis: Biological Insights and Therapeutic Applications.Genome Editing of Erythroid Cell Culture Model Systems.Synergistic effect of two β globin gene cluster mutations leading to the hereditary persistence of fetal hemoglobin (HPFH) phenotype.Does the Novel KLF1 Gene Mutation Lead to a Delay in Fetal Hemoglobin Switch?Genetic disruption of the KLF1 gene to overexpress the γ-globin gene using the CRISPR/Cas9 system.Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing.TRIAMF: 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 Major
P2860
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P2860
Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin.
description
2015 nî lūn-bûn
@nan
2015 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2015年の論文
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2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
Editing the genome to introduc ...... d with increased fetal globin.
@ast
Editing the genome to introduc ...... d with increased fetal globin.
@en
Editing the genome to introduc ...... d with increased fetal globin.
@nl
type
label
Editing the genome to introduc ...... d with increased fetal globin.
@ast
Editing the genome to introduc ...... d with increased fetal globin.
@en
Editing the genome to introduc ...... d with increased fetal globin.
@nl
prefLabel
Editing the genome to introduc ...... d with increased fetal globin.
@ast
Editing the genome to introduc ...... d with increased fetal globin.
@en
Editing the genome to introduc ...... d with increased fetal globin.
@nl
P2093
P2860
P50
P356
P1476
Editing the genome to introduc ...... ed with increased fetal globin
@en
P2093
Alister P W Funnell
Beeke Wienert
Jim Vadolas
Krystal Lester
Lorna E Wilkinson-White
Matthew H Porteus
Richard C M Pearson
P2860
P2888
P356
10.1038/NCOMMS8085
P407
P577
2015-05-14T00:00:00Z
P5875
P6179
1004816533