A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
about
Diagnostic approaches for inherited hemolytic anemia in the genetic eraLong-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.Principles of precision medicine in stroke.Gene Therapy for β-Hemoglobinopathies.Investigational drugs in phase I and phase II clinical trials for thalassemia.Sickle cell disease: tipping the balance of genomic research to catalyse discoveries in Africa.Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia.The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation.Fetal haemoglobin induction in sickle cell disease.Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies.Evolution of hemoglobin loci and their regulatory elements.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.A long noncoding RNA from the HBS1L-MYB intergenic region on chr6q23 regulates human fetal hemoglobin expression.Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage.Comparative analysis of three-dimensional chromosomal architecture identifies a novel fetal hemoglobin regulatory element.Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.Growing and Genetically Manipulating Human Umbilical Cord Blood-Derived Erythroid Progenitor (HUDEP) Cell Lines.Commentary: Programmable base editing of A·T to G·C in genomic DNA without DNA cleavage.Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell Type Specific Gene Editing.Genome Editing for Sickle Cell Disease: A Little BCL11A Goes a Long Way.Generation of App knock-in mice reveals deletion mutations protective against Alzheimer's disease-like pathology.Serum-free Erythroid Differentiation for Efficient Genetic Modification and High-Level Adult Hemoglobin Production.A chance to cut (the genome) is a chance to cure.Efficient Delivery and Nuclear Uptake Is Not Sufficient to Detect Gene Editing in CD34+ Cells Directed by a Ribonucleoprotein Complex.Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies.Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing.CRISPR/Cas9 System: A Bacterial Tailor for Genomic EngineeringTRIAMF: 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
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
description
2016 nî lūn-bûn
@nan
2016年の論文
@ja
2016年学术文章
@wuu
2016年学术文章
@zh
2016年学术文章
@zh-cn
2016年学术文章
@zh-hans
2016年学术文章
@zh-my
2016年学术文章
@zh-sg
2016年學術文章
@yue
2016年學術文章
@zh-hant
name
A genome-editing strategy to t ...... th a benign genetic condition.
@en
A genome-editing strategy to t ...... th a benign genetic condition.
@nl
type
label
A genome-editing strategy to t ...... th a benign genetic condition.
@en
A genome-editing strategy to t ...... th a benign genetic condition.
@nl
prefLabel
A genome-editing strategy to t ...... th a benign genetic condition.
@en
A genome-editing strategy to t ...... th a benign genetic condition.
@nl
P2093
P2860
P50
P356
P1433
P1476
A genome-editing strategy to t ...... ith a benign genetic condition
@en
P2093
Chunliang Li
Elizabeth A Traxler
Gerd A Blobel
Jim R Hughes
Kaitly J Woodard
Ryo Kurita
Yukio Nakamura
P2860
P2888
P304
P356
10.1038/NM.4170
P407
P577
2016-08-15T00:00:00Z