Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.
about
Site-Specific Genome Engineering in Human Pluripotent Stem CellsCustomizing the genome as therapy for the β-hemoglobinopathiesThe therapeutic potential of genome editing for β-thalassemiaSingle-Base Pair Genome Editing in Human Cells by Using Site-Specific EndonucleasesAnemia: progress in molecular mechanisms and therapiesThe potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesTALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicityTALEN based HPV-E7 editing triggers necrotic cell death in cervical cancer cellsNaïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9.Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.Gene therapy for hemoglobinopathies: the state of the field and the future.Genomic editing tools to model human diseases with isogenic pluripotent stem cells.Precision genome editing in plants via gene targeting and piggyBac-mediated marker excisionNuclease-mediated gene editing by homologous recombination of the human globin locus.A single-chain TALEN architecture for genome engineering.Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cellsBoth TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCsProduction of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point MutationGenetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Modulation of human allogeneic and syngeneic pluripotent stem cells and immunological implications for transplantationA genome editing primer for the hematologistCRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.Recent trends for novel options in experimental biological therapy of β-thalassemia.Recent trends in the gene therapy of β-thalassemia.Gene therapy for monogenic disorders of the bone marrow.Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies.Concise Review: Patient-Derived Stem Cell Research for Monogenic Disorders.Induced Pluripotent Stem Cells Derived from Alzheimer's Disease Patients: The Promise, the Hope and the Path Ahead.From hacking the human genome to editing organsModified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies.Current Progress in Therapeutic Gene Editing for Monogenic DiseasesProgrammable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes.Combining Single Strand Oligodeoxynucleotides and CRISPR/Cas9 to Correct Gene Mutations in β-Thalassemia-induced Pluripotent Stem Cells.Gene editing of DNAH11 restores normal cilia motility in primary ciliary dyskinesia.CRISPR-Cas9 technology and its application in haematological disorders.Use of genome-editing tools to treat sickle cell disease.Gene correction in patient-specific iPSCs for therapy development and disease modeling.Silent IL2RG Gene Editing in Human Pluripotent Stem Cells.Emerging cellular and gene therapies for congenital anemias.Nuclease-stimulated homologous recombination at the human β-globin gene.
P2860
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P2860
Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年学术文章
@wuu
2013年学术文章
@zh
2013年学术文章
@zh-cn
2013年学术文章
@zh-hans
2013年学术文章
@zh-my
2013年学术文章
@zh-sg
2013年學術文章
@yue
2013年學術文章
@zh-hant
name
Seamless correction of the sic ...... otent stem cells using TALENs.
@en
Seamless correction of the sic ...... otent stem cells using TALENs.
@nl
type
label
Seamless correction of the sic ...... otent stem cells using TALENs.
@en
Seamless correction of the sic ...... otent stem cells using TALENs.
@nl
prefLabel
Seamless correction of the sic ...... otent stem cells using TALENs.
@en
Seamless correction of the sic ...... otent stem cells using TALENs.
@nl
P2860
P356
P1476
Seamless correction of the sic ...... otent stem cells using TALENs.
@en
P2093
P2860
P304
P356
10.1002/BIT.25018
P577
2013-08-26T00:00:00Z