Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease.
about
RNA-guided human genome engineering via Cas9Site-Specific Genome Engineering in Human Pluripotent Stem CellsHuman induced pluripotent stem cells for monogenic disease modelling and therapyCustomizing the genome as therapy for the β-hemoglobinopathiesGenome-editing Technologies for Gene and Cell TherapyAn Overview of Direct Somatic Reprogramming: The Ins and Outs of iPSCsNeuroprotective therapies in glaucoma: II. Genetic nanotechnology toolsDissecting diabetes/metabolic disease mechanisms using pluripotent stem cells and genome editing toolsProgress and obstacles towards generating hematopoietic stem cells from pluripotent stem cellsThe potential of stem cells as an in vitro source of red blood cells for transfusionSickle cell disease in childrenDisease modeling and cell based therapy with iPSC: future therapeutic option with fast and safe applicationGene therapy on the moveManufacturing blood ex vivo: a futuristic approach to deal with the supply and safety concernsInduced pluripotent stem cells in hematology: current and future applicationsLow-Dose Irradiation Enhances Gene Targeting in Human Pluripotent Stem Cellsβ-Globin sleeping beauty transposon reduces red blood cell sickling in a patient-derived CD34(+)-based in vitro modelTranscription activator-like effector nuclease (TALEN)-mediated CLYBL targeting enables enhanced transgene expression and one-step generation of dual reporter human induced pluripotent stem cell (iPSC) and neural stem cell (NSC) lines.The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesGene therapy for hemophiliaZFN, TALEN, and CRISPR/Cas-based methods for genome engineeringCongenital amegakaryocytic thrombocytopenia iPS cells exhibit defective MPL-mediated signaling.Integration-defective lentiviral vector mediates efficient gene editing through homology-directed repair in human embryonic stem cellsEnhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Differential sensitivity to JAK inhibitory drugs by isogenic human erythroblasts and hematopoietic progenitors generated from patient-specific induced pluripotent stem cellsInduced Pluripotent Stem Cells: Global Research Trends.Differentiation of neuron-like cells from mouse parthenogenetic embryonic stem cells.Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.Human induced pluripotent stem cells can reach complete terminal maturation: in vivo and in vitro evidence in the erythropoietic differentiation model.CRISPR/Cas9 for genome editing: progress, implications and challenges.Investigating human disease using stem cell models.Genomic editing tools to model human diseases with isogenic pluripotent stem cells.Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector.Induced Pluripotent Stem Cells Meet Genome EditingEfficient drug screening and gene correction for treating liver disease using patient-specific stem cells.Stability of zinc finger nuclease protein is enhanced by the proteasome inhibitor MG132.International regulatory landscape and integration of corrective genome editing into in vitro fertilization.Direct cloning of isogenic murine DNA in yeast and relevance of isogenicity for targeting in embryonic stem cells.Generation of 3D skin equivalents fully reconstituted from human induced pluripotent stem cells (iPSCs).Nuclease-mediated gene editing by homologous recombination of the human globin locus.
P2860
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P2860
Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease.
description
2011 nî lūn-bûn
@nan
2011 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Site-specific gene correction ...... ient with sickle cell disease.
@ast
Site-specific gene correction ...... ient with sickle cell disease.
@en
type
label
Site-specific gene correction ...... ient with sickle cell disease.
@ast
Site-specific gene correction ...... ient with sickle cell disease.
@en
prefLabel
Site-specific gene correction ...... ient with sickle cell disease.
@ast
Site-specific gene correction ...... ient with sickle cell disease.
@en
P2093
P2860
P1433
P1476
Site-specific gene correction ...... ient with sickle cell disease.
@en
P2093
Jizhong Zou
Prashant Mali
Sarah N Dowey
Xiaosong Huang
P2860
P304
P356
10.1182/BLOOD-2011-02-335554
P407
P577
2011-08-31T00:00:00Z