Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. - sprookjes - yvandenbroek - TriplyDB

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

http://www.wikidata.org/entity/Q34153816

about

Spermatogonial stem cell autotransplantation and germline genomic editing: a future cure for spermatogenic failure and prevention of transmission of genomic diseases Site-Specific Genome Engineering in Human Pluripotent Stem Cells Customizing the genome as therapy for the β-hemoglobinopathies Genome Engineering with TALE and CRISPR Systems in Neuroscience The Rise of CRISPR/Cas for Genome Editing in Stem Cells iPSCs: A Minireview from Bench to Bed, including Organoids and the CRISPR System The therapeutic potential of genome editing for β-thalassemia Genome-editing Technologies for Gene and Cell Therapy Genome Editing and Its Applications in Model Organisms Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases Single-Base Pair Genome Editing in Human Cells by Using Site-Specific Endonucleases Genome Editing Using Mammalian Haploid Cells Strategies for precision modulation of gene expression by epigenome editing: an overview Advances in CRISPR-Cas9 genome engineering: lessons learned from RNA interference Anemia: progress in molecular mechanisms and therapies Progress and challenges in the development of a cell-based therapy for hemophilia A The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges Survival and Evolution of CRISPR-Cas System in Prokaryotes and Its Applications Exploiting the CRISPR/Cas9 PAM Constraint for Single-Nucleotide Resolution Interventions Enhancement of β-Globin Gene Expression in Thalassemic IVS2-654 Induced Pluripotent Stem Cell-Derived Erythroid Cells by Modified U7 snRNA.Induced Pluripotent Stem Cells: Global Research Trends.Naïve Induced Pluripotent Stem Cells Generated From β-Thalassemia Fibroblasts Allow Efficient Gene Correction With CRISPR/Cas9.Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells CRISPR/Cas9 in allergic and immunologic diseases.What is CRISPR/Cas9?Induced Pluripotent Stem Cells Meet Genome Editing Applications of CRISPR technologies in research and beyond.Eliminate mitochondrial diseases by gene editing in germ-line cells and embryos.Both TALENs and CRISPR/Cas9 directly target the HBB IVS2-654 (C > T) mutation in β-thalassemia-derived iPSCs CRISPR-Cas9-mediated gene knockout in primary human airway epithelial cells reveals a proinflammatory role for MUC18.Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation Pluripotent stem cell applications for regenerative medicine Comparative genomics of canine hemoglobin genes reveals primacy of beta subunit delta in adult carnivores.The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice.Co-incident insertion enables high efficiency genome engineering in mouse embryonic stem cells.Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia CRISPR RNA-guided FokI nucleases repair a PAH variant in a phenylketonuria model.CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.Concise Review: Getting to the Core of Inherited Bone Marrow Failures.Proteasome impairment in neural cells derived from HMSN-P patient iPSCs.

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Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

http://www.wikidata.org/entity/Q34153816

description

2014 nî lūn-bûn

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2014 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

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2014 թվականի օգոստոսին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Seamless gene correction of β- ...... sing CRISPR/Cas9 and piggyBac.

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Ashley I Beyer

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Fei Xie

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Jiaming Wang

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Judy C Chang

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Lin Ye

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Yuet Wai Kan

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P2860

RNA-guided human genome engineering via Cas9

Multiplex genome engineering using CRISPR/Cas systems

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Gene therapy: therapeutic gene causing lymphoma

Targeted genome editing across species using ZFNs and TALENs

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

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1526-1533

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10.1101/GR.173427.114

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9

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24

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Marcus O. Muench

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25096406

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4158758

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