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Customizing the genome as therapy for the β-hemoglobinopathiesUnderstanding Spatial Genome Organization: Methods and InsightsGenome-editing Technologies for Gene and Cell TherapyMaking sense of GWAS: using epigenomics and genome engineering to understand the functional relevance of SNPs in non-coding regions of the human genomeTowards a new era in medicine: therapeutic genome editingFunctional interrogation of non-coding DNA through CRISPR genome editing.CRISPR-Cas9 epigenome editing enables high-throughput screening for functional regulatory elements in the human genome.Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition.Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous diseaseRegulation of the fetal hemoglobin silencing factor BCL11A.Recent advances in globin research using genome-wide association studies and gene editing.A genome editing primer for the hematologistInheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic EditingCRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.Current Progress in Therapeutic Gene Editing for Monogenic DiseasesSmall Genetic Circuits and MicroRNAs: Big Players in Polymerase II Transcriptional Control in Plants.Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.Precision medicine, genomics and drug discovery.Emerging cellular and gene therapies for congenital anemias.Cornerstones of CRISPR-Cas in drug discovery and therapy.Transcription factor-DNA binding: beyond binding site motifs.Gene Therapy for β-Hemoglobinopathies.Molecular basis of β thalassemia and potential therapeutic targets.Recent advances in high-throughput approaches to dissect enhancer functionThe epigenetic landscape of T cell exhaustion.High-throughput mapping of regulatory DNA.Genome-scale high-resolution mapping of activating and repressive nucleotides in regulatory regions.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.Complex relationships between chromatin accessibility, sequence divergence, and gene expression in A. thaliana.Pathophysiology and treatment of patients with beta-thalassemia - an update.A CRISPR view of gene regulation.CRISPR-based reagents to study the influence of the epigenome on gene expressionDisruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia MajorGene Therapy for Hemoglobinopathies
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P2860
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on 31 August 2015
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Functional footprinting of regulatory DNA.
@en
Functional footprinting of regulatory DNA.
@nl
type
label
Functional footprinting of regulatory DNA.
@en
Functional footprinting of regulatory DNA.
@nl
prefLabel
Functional footprinting of regulatory DNA.
@en
Functional footprinting of regulatory DNA.
@nl
P2093
P2860
P356
P1433
P1476
Functional footprinting of regulatory DNA
@en
P2093
Alexander H Song
Andrea K Mich
Andreas Reik
Colleen M O'Neil
Daniel E Bauer
David E Paschon
Edward J Rebar
Fyodor D Urnov
George Stamatoyannopoulos
P2860
P2888
P304
P356
10.1038/NMETH.3554
P577
2015-08-31T00:00:00Z