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Identification of preexisting adaptive immunity to Cas9 proteins in humans

Nature medicine, 2019-02, Vol.25 (2), p.249-254

2019

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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Nature medicine, 2018-08, Vol.24 (8), p.1216-1224

2018

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The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation

Current opinion in hematology, 2017-11, Vol.24 (6), p.481-488

2017

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Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

Nature communications, 2021-01, Vol.12 (1), p.686-686, Article 686

2021

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Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

Cell stem cell, 2019-05, Vol.24 (5), p.821-828.e5

2019

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Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells

Molecular therapy, 2018-10, Vol.26 (10), p.2431-2442

2018

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The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

Nature communications, 2021-01, Vol.12 (1), p.472-472, Article 472

2021

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Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

eLife, 2017-09, Vol.6

2017

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Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

Molecular therapy. Nucleic acids, 2018-09, Vol.12, p.89-104

2018

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Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells

Nature communications, 2024-01, Vol.15 (1), p.111-111, Article 111

2024

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CRISPR/Cas9 genome editing in human hematopoietic stem cells

Nature protocols, 2018-02, Vol.13 (2), p.358-376

2018

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Aryl hydrocarbon receptor deletion in cerebellar granule neuron precursors impairs neurogenesis

Developmental neurobiology (Hoboken, N.J.), 2016-05, Vol.76 (5), p.533-550

2016

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Technical considerations for the use of CRISPR/Cas9 in hematology research

Experimental hematology, 2017-10, Vol.54, p.4-11

2017

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Nature (London), 2016-11, Vol.539 (7629), p.384-389

2016

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Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency

Molecular therapy. Methods & clinical development, 2021-09, Vol.22, p.237-248

2021

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CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells

iScience, 2019-05, Vol.15, p.524-535

2019

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Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Nucleic acids research, 2019-09, Vol.47 (15), p.7955

2019

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2,3,7,8-Tetracholorodibenzo-p-Dioxin Exposure Disrupts Granule Neuron Precursor Maturation in the Developing Mouse Cerebellum

Toxicological sciences, 2008-05, Vol.103 (1), p.125-136

2008

Volltextzugriff (PDF)

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Science translational medicine, 2021-06, Vol.13 (598)

2021

Volltextzugriff (PDF)

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Nature medicine, 2021-04, Vol.27 (4), p.677-687

2021

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The aryl hydrocarbon receptor contributes to the proliferation of human medulloblastoma cells

Molecular pharmacology, 2012-05, Vol.81 (5), p.669-678

2012

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Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair

Nature communications, 2024-03, Vol.15 (1), p.2625-2625, Article 2625

2024

Volltextzugriff (PDF)

Highly Efficient Editing of the Beta-Globin Gene in Patient Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease

Blood, 2018-11, Vol.132 (Supplement 1), p.2192-2192

2018

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Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

eLife, 2018-11, Vol.7

2018

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Subchronic Polychlorinated Biphenyl (Aroclor 1254) Exposure Produces Oxidative Damage and Neuronal Death of Ventral Midbrain Dopaminergic Systems

Toxicological sciences, 2012-02, Vol.125 (2), p.496-508

2012

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Identification of preexisting adaptive immunity to Cas9 proteins in humans

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells

CRISPR/Cas9 genome editing in human hematopoietic stem cells

Aryl hydrocarbon receptor deletion in cerebellar granule neuron precursors impairs neurogenesis

Technical considerations for the use of CRISPR/Cas9 in hematology research

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency

CRISPR/Cas9 Genome Engineering in Engraftable Human Brain-Derived Neural Stem Cells

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

2,3,7,8-Tetracholorodibenzo-p-Dioxin Exposure Disrupts Granule Neuron Precursor Maturation in the Developing Mouse Cerebellum

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

The aryl hydrocarbon receptor contributes to the proliferation of human medulloblastoma cells

Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair

Highly Efficient Editing of the Beta-Globin Gene in Patient Derived Hematopoietic Stem and Progenitor Cells to Treat Sickle Cell Disease

Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6

Subchronic Polychlorinated Biphenyl (Aroclor 1254) Exposure Produces Oxidative Damage and Neuronal Death of Ventral Midbrain Dopaminergic Systems

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