Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs
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CRISPR/Cas9 therapeutics: a cure for cancer and other genetic diseasesHuman induced pluripotent stem cells for monogenic disease modelling and therapyThe Rise of CRISPR/Cas for Genome Editing in Stem CellsThe potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesIn vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle deliveryEnhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Applications of CRISPR technologies in research and beyond.High-frequency, precise modification of the tomato genome.Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.TALENs Facilitate Single-step Seamless SDF Correction of F508del CFTR in Airway Epithelial Submucosal Gland Cell-derived CF-iPSCs.Analyses of point mutation repair and allelic heterogeneity generated by CRISPR/Cas9 and single-stranded DNA oligonucleotides.Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemiaConcise Review: Getting to the Core of Inherited Bone Marrow Failures.One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection.Current Progress in Therapeutic Gene Editing for Monogenic DiseasesCRISPR/Cas9-Directed Reassignment of the GATA1 Initiation Codon in K562 Cells to Recapitulate AML in Down Syndrome.Pluripotent stem cells progressing to the clinic.Programmable 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.CD34+ cells from dental pulp stem cells with a ZFN-mediated and homology-driven repair-mediated locus-specific knock-in of an artificial β-globin gene.CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.Generation of improved human cerebral organoids from single copy DYRK1A knockout induced pluripotent stem cells in trisomy 21: hypothetical solutions for neurodevelopmental models and therapeutic alternatives in down syndrome.CRISPR-Cas9 technology and its application in haematological disorders.A comprehensive overview of computational resources to aid in precision genome editing with engineered nucleases.Therapeutic applications of CRISPR RNA-guided genome editing.Emerging cellular and gene therapies for congenital anemias.Cure for thalassemia major - from allogeneic hematopoietic stem cell transplantation to gene therapy.Precision genome editing in the CRISPR era.Therapeutic gene editing: delivery and regulatory perspectivesInvestigational drugs in phase I and phase II clinical trials for thalassemia.Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia.Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing.A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.Emerging Therapies.One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system.Deoxyribonucleic Acid Damage and Repair: Capitalizing on Our Understanding of the Mechanisms of Maintaining Genomic Integrity for Therapeutic Purposes.Efficient Delivery and Nuclear Uptake Is Not Sufficient to Detect Gene Editing in CD34+ Cells Directed by a Ribonucleoprotein Complex.Erythropoiesis: insights into pathophysiology and treatments in 2017.CRISPR-mediated genome editing and human diseases
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P2860
Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
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
2015年论文
@zh
2015年论文
@zh-cn
name
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@ast
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@en
type
label
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@ast
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@en
prefLabel
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@ast
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@en
P2093
P2860
P356
P1433
P1476
Both TALENs and CRISPR/Cas9 di ...... in β-thalassemia-derived iPSCs
@en
P2093
Bo-yu Wang
De-pei Liu
Ting-ting Wang
Xiu-zhen Liu
P2860
P2888
P356
10.1038/SREP12065
P407
P50
P577
2015-07-09T00:00:00Z