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Gene therapy comes of age

Science (American Association for the Advancement of Science), 2018-01, Vol.359 (6372)

2018

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Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles

Human gene therapy, 2016-10, Vol.27 (10), p.729

2016

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An Introduction to the Analysis of Single-Cell RNA-Sequencing Data

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.189-196

2018

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Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

Frontiers of medicine, 2011-12, Vol.5 (4), p.356-371

2011

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Clonal Tracking of Rhesus Macaque Hematopoiesis Highlights a Distinct Lineage Origin for Natural Killer Cells

Cell stem cell, 2014-04, Vol.14 (4), p.486-499

2014

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Dynamics of HSPC Repopulation in Nonhuman Primates Revealed by a Decade-Long Clonal-Tracking Study

Cell stem cell, 2014-04, Vol.14 (4), p.473-485

2014

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Predictors of clonal evolution and myeloid neoplasia following immunosuppressive therapy in severe aplastic anemia

Leukemia, 2022-09, Vol.36 (9), p.2328-2337

2022

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Genotoxicity of Retroviral Integration In Hematopoietic Cells

Molecular therapy, 2006-06, Vol.13 (6), p.1031-1049

2006

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Applying a clinical lens to animal models of CAR-T cell therapies

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.17-31

2022

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Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model

Cell reports (Cambridge), 2014-05, Vol.7 (4), p.1298-1309

2014

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Assessing the Risks of Genotoxicity in the Therapeutic Development of Induced Pluripotent Stem Cells

Molecular therapy, 2013-02, Vol.21 (2), p.272-281

2013

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iPSCs and fibroblast subclones from the same fibroblast population contain comparable levels of sequence variations

Proceedings of the National Academy of Sciences - PNAS, 2017-02, Vol.114 (8), p.1964-1969

2017

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MRI Detection of Single Particles for Cellular Imaging

Proceedings of the National Academy of Sciences - PNAS, 2004-07, Vol.101 (30), p.10901-10906

2004

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Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Nature communications, 2023-10, Vol.14 (1), p.6291-6291, Article 6291

2023

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Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome

Haematologica (Roma), 2020-12, Vol.105 (12), p.2785-2794

2020

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Modeling Human Bone Marrow Failure Syndromes Using Pluripotent Stem Cells and Genome Engineering

Molecular therapy, 2015-12, Vol.23 (12), p.1832-1842

2015

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Functional Niche Competition Between Normal Hematopoietic Stem and Progenitor Cells and Myeloid Leukemia Cells

Stem cells (Dayton, Ohio), 2015-12, Vol.33 (12), p.3635-3642

2015

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Acquired somatic mutations in PNH reveal long-term maintenance of adaptive NK cells independent of HSPCs

Blood, 2017-04, Vol.129 (14), p.1940-1946

2017

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A plethora of gene therapies for hemoglobinopathies

Nature medicine, 2021-02, Vol.27 (2), p.202-204

Dunbar, Cynthia E

2021

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The Role of Nonhuman Primate Animal Models in the Clinical Development of Pluripotent Stem Cell Therapies

Molecular therapy, 2016-07, Vol.24 (7), p.1165-1169

2016

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Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

Molecular therapy, 2012-07, Vol.20 (7), p.1410-1416

2012

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Tissue Trafficking Kinetics of Rhesus Macaque Natural Killer Cells Measured by Serial Intravascular Staining

Frontiers in immunology, 2022-01, Vol.12, p.772332-772332

2022

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Defective telomere elongation and hematopoiesis from telomerase-mutant aplastic anemia iPSCs

The Journal of clinical investigation, 2013-05, Vol.123 (5), p.1952-1963

2013

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Transcriptome analysis reveals similarities between human blood CD3− CD56bright cells and mouse CD127+ innate lymphoid cells

Scientific reports, 2017-06, Vol.7 (1), p.1-11, Article 3501

2017

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Persistent elevation of plasma thrombopoietin levels after treatment in severe aplastic anemia

Experimental hematology, 2018-02, Vol.58, p.39-43

2018

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Gene therapy comes of age

Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles

An Introduction to the Analysis of Single-Cell RNA-Sequencing Data

Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

Clonal Tracking of Rhesus Macaque Hematopoiesis Highlights a Distinct Lineage Origin for Natural Killer Cells

Dynamics of HSPC Repopulation in Nonhuman Primates Revealed by a Decade-Long Clonal-Tracking Study

Predictors of clonal evolution and myeloid neoplasia following immunosuppressive therapy in severe aplastic anemia

Genotoxicity of Retroviral Integration In Hematopoietic Cells

Applying a clinical lens to animal models of CAR-T cell therapies

Path to the Clinic: Assessment of iPSC-Based Cell Therapies In Vivo in a Nonhuman Primate Model

Assessing the Risks of Genotoxicity in the Therapeutic Development of Induced Pluripotent Stem Cells

iPSCs and fibroblast subclones from the same fibroblast population contain comparable levels of sequence variations

MRI Detection of Single Particles for Cellular Imaging

Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model

Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome

Modeling Human Bone Marrow Failure Syndromes Using Pluripotent Stem Cells and Genome Engineering

Functional Niche Competition Between Normal Hematopoietic Stem and Progenitor Cells and Myeloid Leukemia Cells

Acquired somatic mutations in PNH reveal long-term maintenance of adaptive NK cells independent of HSPCs

A plethora of gene therapies for hemoglobinopathies

The Role of Nonhuman Primate Animal Models in the Clinical Development of Pluripotent Stem Cell Therapies

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B

Tissue Trafficking Kinetics of Rhesus Macaque Natural Killer Cells Measured by Serial Intravascular Staining

Defective telomere elongation and hematopoiesis from telomerase-mutant aplastic anemia iPSCs

Transcriptome analysis reveals similarities between human blood CD3− CD56bright cells and mouse CD127+ innate lymphoid cells

Persistent elevation of plasma thrombopoietin levels after treatment in severe aplastic anemia

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