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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Science translational medicine, 2016-04, Vol.8 (335), p.335ra57

2016

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Clinical strategies for expansion of haematopoietic stem cells

Nature reviews. Immunology, 2004-11, Vol.4 (11), p.878-888

Sorrentino, Brian P

2004

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Drug transporters on arachnoid barrier cells contribute to the blood-cerebrospinal fluid barrier

Drug metabolism and disposition, 2013-04, Vol.41 (4), p.923

2013

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The Stem Cell Marker Bcrp/ABCG2 Enhances Hypoxic Cell Survival through Interactions with Heme

The Journal of biological chemistry, 2004-06, Vol.279 (23), p.24218-24225

2004

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Abcg2 Expression Marks Tissue-Specific Stem Cells in Multiple Organs in a Mouse Progeny Tracking Model

Stem cells (Dayton, Ohio), 2012-02, Vol.30 (2), p.210-221

2012

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Cells previously identified as retinal stem cells are pigmented ciliary epithelial cells

Proceedings of the National Academy of Sciences - PNAS, 2009-04, Vol.106 (16), p.6685-6690

2009

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The ABC transporter Bcrp1/ABCG2 is expressed in a wide variety of stem cells and is a molecular determinant of the side-population phenotype

Nature medicine, 2001-09, Vol.7 (9), p.1028-1034

2001

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Bcrp1 Gene Expression Is Required for Normal Numbers of Side Population Stem Cells in Mice, and Confers Relative Protection to Mitoxantrone in Hematopoietic Cells in vivo

Proceedings of the National Academy of Sciences - PNAS, 2002-09, Vol.99 (19), p.12339-12344

2002

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Unique Risk Factors for Insertional Mutagenesis in a Mouse Model of XSCID Gene Therapy

Proceedings of the National Academy of Sciences - PNAS, 2006-08, Vol.103 (31), p.11730-11735

2006

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Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

Blood, 2009-05, Vol.113 (21), p.5104-5110

2009

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ABCG2 Transporter Expression Impacts Group 3 Medulloblastoma Response to Chemotherapy

Cancer research (Chicago, Ill.), 2015-09, Vol.75 (18), p.3879-3889

2015

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Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector

PloS one, 2013-04, Vol.8 (4), p.e62333

2013

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Increased expression of the Abcg2 transporter during erythroid maturation plays a role in decreasing cellular protoporphyrin IX levels

Blood, 2005-03, Vol.105 (6), p.2571-2576

2005

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3’ UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates

Molecular therapy. Methods & clinical development, 2021-06, Vol.21, p.693-701

2021

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Upregulated heme biosynthesis, an exploitable vulnerability in MYCN-driven leukemogenesis

JCI insight, 2017-08, Vol.2 (15)

2017

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Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

The New England journal of medicine, 2019-04, Vol.380 (16), p.1525-1534

2019

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Hemgn is a direct transcriptional target of HOXB4 and induces expansion of murine myeloid progenitor cells

Blood, 2010-08, Vol.116 (5), p.711-719

2010

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Marking of definitive HSC precursors in E7.5–E8.5 embryos using an Abcg2-CreER lineage-tracing mouse model

Experimental hematology, 2018-09, Vol.65, p.29-33

2018

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Low levels of ABCG2 expression in adult AML blast samples

Blood, 2002-12, Vol.100 (13), p.4594-4601

2002

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Function-dependent Conformational Changes of the ABCG2 Multidrug Transporter Modify Its Interaction with a Monoclonal Antibody on the Cell Surface

The Journal of biological chemistry, 2005-02, Vol.280 (6), p.4219-4227

2005

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Interleukin-7 receptor mutants initiate early T cell precursor leukemia in murine thymocyte progenitors with multipotent potential

The Journal of experimental medicine, 2014-04, Vol.211 (4), p.701-713

2014

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Expression of Mouse Abcg2 mRNA during Hematopoiesis Is Regulated by Alternative Use of Multiple Leader Exons and Promoters

The Journal of biological chemistry, 2006-10, Vol.281 (40), p.29625-29632

2006

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Interaction with the 5D3 Monoclonal Antibody Is Regulated by Intramolecular Rearrangements but Not by Covalent Dimer Formation of the Human ABCG2 Multidrug Transporter

The Journal of biological chemistry, 2008-09, Vol.283 (38), p.26059-26070

2008

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Prospective Isolation of Murine Hematopoietic Stem Cells by Expression of an Abcg2/GFP Allele

Stem cells (Dayton, Ohio), 2006-06, Vol.24 (6), p.1556-1563

2006

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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Clinical strategies for expansion of haematopoietic stem cells

Drug transporters on arachnoid barrier cells contribute to the blood-cerebrospinal fluid barrier

The Stem Cell Marker Bcrp/ABCG2 Enhances Hypoxic Cell Survival through Interactions with Heme

Abcg2 Expression Marks Tissue-Specific Stem Cells in Multiple Organs in a Mouse Progeny Tracking Model

Cells previously identified as retinal stem cells are pigmented ciliary epithelial cells

The ABC transporter Bcrp1/ABCG2 is expressed in a wide variety of stem cells and is a molecular determinant of the side-population phenotype

Bcrp1 Gene Expression Is Required for Normal Numbers of Side Population Stem Cells in Mice, and Confers Relative Protection to Mitoxantrone in Hematopoietic Cells in vivo

Unique Risk Factors for Insertional Mutagenesis in a Mouse Model of XSCID Gene Therapy

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

ABCG2 Transporter Expression Impacts Group 3 Medulloblastoma Response to Chemotherapy

Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector

Increased expression of the Abcg2 transporter during erythroid maturation plays a role in decreasing cellular protoporphyrin IX levels

The genomic landscape of pediatric and young adult T-lineage acute lymphoblastic leukemia

3’ UTR-truncated HMGA2 overexpression induces non-malignant in vivo expansion of hematopoietic stem cells in non-human primates

Upregulated heme biosynthesis, an exploitable vulnerability in MYCN-driven leukemogenesis

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Hemgn is a direct transcriptional target of HOXB4 and induces expansion of murine myeloid progenitor cells

Marking of definitive HSC precursors in E7.5–E8.5 embryos using an Abcg2-CreER lineage-tracing mouse model

Low levels of ABCG2 expression in adult AML blast samples

Function-dependent Conformational Changes of the ABCG2 Multidrug Transporter Modify Its Interaction with a Monoclonal Antibody on the Cell Surface

Interleukin-7 receptor mutants initiate early T cell precursor leukemia in murine thymocyte progenitors with multipotent potential

Expression of Mouse Abcg2 mRNA during Hematopoiesis Is Regulated by Alternative Use of Multiple Leader Exons and Promoters

Interaction with the 5D3 Monoclonal Antibody Is Regulated by Intramolecular Rearrangements but Not by Covalent Dimer Formation of the Human ABCG2 Multidrug Transporter

Prospective Isolation of Murine Hematopoietic Stem Cells by Expression of an Abcg2/GFP Allele

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