Publications by authors named "Paul Gadue"

67Publications

Generation of a double insulin and somatostatin reporter line, SCSe001-A-3, for the advancement of stem cell-derived pancreatic islets.

Stem Cell Res 2020 Dec 8;50:102112. Epub 2020 Dec 8.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, United States. Electronic address:

View Article and Find Full Text PDF
December 2020

Large-scale Generation of Functional and Transplantable Hepatocytes and Cholangiocytes from Human Endoderm Stem Cells.

Cell Rep 2020 Dec;33(10):108455

State Key Laboratory of Cell Biology, CAS Center for Excellence in Molecular Cell Science, Institute of Biochemistry and Cell Biology, University of Chinese Academy of Sciences, Chinese Academy of Sciences, 200031 Shanghai, China. Electronic address:

View Article and Find Full Text PDF
December 2020

Generation of human control iPSC line CHOPi004-A from juvenile foreskin fibroblast cells.

Stem Cell Res 2020 Dec 7;49:102084. Epub 2020 Nov 7.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, USA; Department of Pathology and Laboratory Medicine, University of Pennsylavania, USA. Electronic address:

View Article and Find Full Text PDF
December 2020

A Non-Coding Disease Modifier of Pancreatic Agenesis Identified by Genetic Correction in a Patient-Derived iPSC Line.

Cell Stem Cell 2020 Jul 21;27(1):137-146.e6. Epub 2020 May 21.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address:

View Article and Find Full Text PDF
July 2020

Tropomyosin 1 genetically constrains in vitro hematopoiesis.

BMC Biol 2020 05 14;18(1):52. Epub 2020 May 14.

Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

View Article and Find Full Text PDF
May 2020

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells.

J Vis Exp 2019 09 25(151). Epub 2019 Sep 25.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia; Department of Pathology and Laboratory Medicine, University of Pennsylvania;

View Article and Find Full Text PDF
September 2019

Modeling Monogenic Diabetes using Human ESCs Reveals Developmental and Metabolic Deficiencies Caused by Mutations in HNF1A.

Cell Stem Cell 2019 08;25(2):273-289.e5

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address:

View Article and Find Full Text PDF
August 2019

Generation of Spinocerebellar Ataxia Type 2 induced pluripotent stem cell lines, CHOPi002-A and CHOPi003-A, from patients with abnormal CAG repeats in the coding region of the ATXN2 gene.

Stem Cell Res 2019 01 10;34:101361. Epub 2018 Dec 10.

Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia and The University of Pennsylvania, USA. Electronic address:

View Article and Find Full Text PDF
January 2019

Highly Efficient CRISPR-Cas9-Mediated Genome Editing in Human Pluripotent Stem Cells.

Curr Protoc Stem Cell Biol 2019 02 24;48(1):e64. Epub 2018 Oct 24.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

View Article and Find Full Text PDF
February 2019

Generation of a human Juvenile myelomonocytic leukemia iPSC line, CHOPi001-A, with a mutation in CBL.

Stem Cell Res 2018 08 5;31:157-160. Epub 2018 Jul 5.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, United States. Electronic address:

View Article and Find Full Text PDF
August 2018

Exploring the Interaction Between eIF2α Dysregulation, Acute Endoplasmic Reticulum Stress and DYT1 Dystonia in the Mammalian Brain.

Neuroscience 2018 02 28;371:455-468. Epub 2017 Dec 28.

Raymond G. Perelman Center for Cellular & Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, United States; Department of Neurology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, United States. Electronic address:

View Article and Find Full Text PDF
February 2018

Induced Pluripotent Stem Cell-Derived Megakaryocytes and Platelets for Disease Modeling and Future Clinical Applications.

Arterioscler Thromb Vasc Biol 2017 11 5;37(11):2007-2013. Epub 2017 Oct 5.

From the Department of Cell and Molecular Biology, Perelman School of Medicine (S.B., X.S.), Department of Pharmacology, Perelman School of Medicine (M.P.), and Department of Pathology and Laboratory Medicine (D.L.F., P.G.), University of Pennsylvania, Philadelphia; and Center for Cellular and Molecular Therapeutics (S.B., X.S., D.L.F., P.G.) and Division of Hematology (M.P.), Children's Hospital of Philadelphia, PA.

View Article and Find Full Text PDF
November 2017

Identifying and enriching platelet-producing human stem cell-derived megakaryocytes using factor V uptake.

Blood 2017 07 28;130(2):192-204. Epub 2017 Apr 28.

Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA.

View Article and Find Full Text PDF
July 2017

GATA6 Plays an Important Role in the Induction of Human Definitive Endoderm, Development of the Pancreas, and Functionality of Pancreatic β Cells.

Stem Cell Reports 2017 03 9;8(3):589-604. Epub 2017 Feb 9.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, CTRB 5012, 3501 Civic Center Boulevard, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address:

View Article and Find Full Text PDF
March 2017

Generation of human control iPS cell line CHOPWT10 from healthy adult peripheral blood mononuclear cells.

Stem Cell Res 2016 Mar 3;16(2):338-41. Epub 2016 Feb 3.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, University of Pennsylvania, United States.

View Article and Find Full Text PDF
March 2016

Generation of Hermansky Pudlak syndrome type 2 (HPS2) induced pluripotent stem cells (iPSCs).

Stem Cell Res 2016 Mar 14;16(2):287-9. Epub 2016 Jan 14.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, United States; University of Pennsylvania, United States.

View Article and Find Full Text PDF
March 2016

Generation of Hermansky-Pudlak Syndrome Type 1 (HPS1) induced pluripotent stem cells (iPSCs).

Stem Cell Res 2016 Mar 14;16(2):233-5. Epub 2016 Jan 14.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, and University of Pennsylvania, United States.

View Article and Find Full Text PDF
March 2016

Generation of human control iPS cell line CHOPWT9 from healthy adult peripheral blood mononuclear cells.

Stem Cell Res 2016 Jan 19;16(1):14-6. Epub 2015 Nov 19.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, United States; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, United States; University of Pennsylvania, United States.

View Article and Find Full Text PDF
January 2016

Generation of poikiloderma with neutropenia (PN) induced pluripotent stem cells (iPSCs).

Stem Cell Res 2015 Nov 16;15(3):595-7. Epub 2015 Oct 16.

Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, The University of Pennsylvania, Philadelphia, PA, USA.

View Article and Find Full Text PDF
November 2015

Factors Regulating Stem Cell Biology in Development and Disease.

Stem Cells Int 2016 20;2016:7170642. Epub 2015 Dec 20.

Department of Internal Medicine I, University of Ulm, Albert-Einstein-Allee 11, 89081 Ulm, Germany.

View Article and Find Full Text PDF
January 2016

Understanding platelet generation from megakaryocytes: implications for in vitro-derived platelets.

Blood 2016 Mar 19;127(10):1227-33. Epub 2016 Jan 19.

Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA; Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia PA; and Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA.

View Article and Find Full Text PDF
March 2016

OCT4 Coordinates with WNT Signaling to Pre-pattern Chromatin at the SOX17 Locus during Human ES Cell Differentiation into Definitive Endoderm.

Stem Cell Reports 2015 Oct 24;5(4):490-8. Epub 2015 Sep 24.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address:

View Article and Find Full Text PDF
October 2015

Comparative analysis of human ex vivo-generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale.

Blood 2015 Jun 7;125(23):3627-36. Epub 2015 Apr 7.

Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA; Departments of Pediatrics and Pathology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA;

View Article and Find Full Text PDF
June 2015

A Doxycycline-Inducible System for Genetic Correction of iPSC Disease Models.

Methods Mol Biol 2016 ;1353:13-23

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.

View Article and Find Full Text PDF
August 2016

Dynamics of genomic H3K27me3 domains and role of EZH2 during pancreatic endocrine specification.

EMBO J 2014 Oct 8;33(19):2157-70. Epub 2014 Aug 8.

Institute for Regenerative Medicine, Epigenetics Program, Department of Cell and Developmental Biology, Perelman School of Medicine University of Pennsylvania, Philadelphia, PA, USA

View Article and Find Full Text PDF
October 2014

Hematopoietic differentiation of pluripotent stem cells in culture.

Methods Mol Biol 2014 ;1185:181-94

Department of Pathology and Laboratory Medicine and Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.

View Article and Find Full Text PDF
March 2015

Utilization of the AAVS1 safe harbor locus for hematopoietic specific transgene expression and gene knockdown in human ES cells.

Stem Cell Res 2014 May 21;12(3):630-7. Epub 2014 Feb 21.

Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. Electronic address:

View Article and Find Full Text PDF
May 2014

MicroRNA screen of human embryonic stem cell differentiation reveals miR-105 as an enhancer of megakaryopoiesis from adult CD34+ cells.

Stem Cells 2014 May;32(5):1337-46

Institute for Medicine and Engineering, Department of Chemical and Biomolecular Engineering, 1024 Vagelos Research Laboratory, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

View Article and Find Full Text PDF
May 2014

The negative impact of Wnt signaling on megakaryocyte and primitive erythroid progenitors derived from human embryonic stem cells.

Stem Cell Res 2014 Mar 16;12(2):441-51. Epub 2013 Dec 16.

Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia, PA, USA. Electronic address:

View Article and Find Full Text PDF
March 2014

AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

PLoS One 2013 7;8(5):e61396. Epub 2013 May 7.

F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, United States of America.

View Article and Find Full Text PDF
December 2013

Endodermal stem cell populations derived from pluripotent stem cells.

Curr Opin Cell Biol 2013 Apr 26;25(2):265-71. Epub 2013 Feb 26.

Center for Cellular and Molecular Therapeutics, and Department of Pathology & Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA, USA.

View Article and Find Full Text PDF
April 2013

Foxa2 and H2A.Z mediate nucleosome depletion during embryonic stem cell differentiation.

Cell 2012 Dec;151(7):1608-16

Department of Genetics and Institute of Diabetes, Obesity and Metabolism, School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

View Article and Find Full Text PDF
December 2012

High-throughput screening assay for embryoid body differentiation of human embryonic stem cells.

Curr Protoc Stem Cell Biol 2012 Mar;Chapter 1:Unit 1D.6

Institute for Medicine and Engineering, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

View Article and Find Full Text PDF
March 2012

An endothelial cell niche induces hepatic specification through dual repression of Wnt and Notch signaling.

Stem Cells 2011 Feb;29(2):217-28

Department of Gene and Cell Medicine, Black Family Stem Cell Institute, Mount Sinai School of Medicine, New York, New York 10029, USA.

View Article and Find Full Text PDF
February 2011

A high-throughput multiplexed screening assay for optimizing serum-free differentiation protocols of human embryonic stem cells.

Stem Cell Res 2011 Mar 16;6(2):129-42. Epub 2010 Nov 16.

Institute for Medicine and Engineering, University of Pennsylvania, Philadelphia, PA 19104, USA.

View Article and Find Full Text PDF
March 2011

Liver regeneration from induced pluripotent stem cells.

Mol Ther 2010 Dec;18(12):2044-5

Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, USA.

View Article and Find Full Text PDF
December 2010

Transcriptional competence and the active marking of tissue-specific enhancers by defined transcription factors in embryonic and induced pluripotent stem cells.

Genes Dev 2009 Dec;23(24):2824-38

Molecular Biology Institute, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California at Los Angeles, Los Angeles, California 90095, USA.

View Article and Find Full Text PDF
December 2009

Stem cells unscramble yolk sac hematopoiesis.

Blood 2009 Aug;114(8):1455-6

The Children's Hospital of Philadelphia, USA.

View Article and Find Full Text PDF
August 2009

Generation of monoclonal antibodies specific for cell surface molecules expressed on early mouse endoderm.

Stem Cells 2009 Sep;27(9):2103-13

Department of Pathology, Center for Cellular and Molecular Therapeutics; Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

View Article and Find Full Text PDF
September 2009

Numb mediates the interaction between Wnt and Notch to modulate primitive erythropoietic specification from the hemangioblast.

Development 2008 Oct 17;135(20):3447-58. Epub 2008 Sep 17.

Department of Gene and Cell Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA.

View Article and Find Full Text PDF
October 2008

Wnt, activin, and BMP signaling regulate distinct stages in the developmental pathway from embryonic stem cells to blood.

Cell Stem Cell 2008 Jan;2(1):60-71

McEwen Centre for Regenerative Medicine, University Health Network, Toronto, ON M5G 1L7, Canada.

View Article and Find Full Text PDF
January 2008

Identification and targeting of the ROSA26 locus in human embryonic stem cells.

Nat Biotechnol 2007 Dec 25;25(12):1477-82. Epub 2007 Nov 25.

University Health Network, McEwen Centre for Regenerative Medicine, 101 College Street, Toronto, ON, M5G 1L7, Canada.

View Article and Find Full Text PDF
December 2007

BMP-4 is required for hepatic specification of mouse embryonic stem cell-derived definitive endoderm.

Nat Biotechnol 2006 Nov 5;24(11):1402-11. Epub 2006 Nov 5.

Department of Gene and Cell Medicine, Black Family Stem Cell Institute, Mount Sinai School of Medicine, 1425 Madison Avenue, New York, New York 10029, USA.

View Article and Find Full Text PDF
November 2006

Wnt and TGF-beta signaling are required for the induction of an in vitro model of primitive streak formation using embryonic stem cells.

Proc Natl Acad Sci U S A 2006 Nov 31;103(45):16806-11. Epub 2006 Oct 31.

Department of Gene and Cell Medicine, Black Family Stem Cell Institute, Mount Sinai School of Medicine, 1 Gustave Levy Place, Box 1496, New York, NY 10029, USA.

View Article and Find Full Text PDF
November 2006

Germ layer induction from embryonic stem cells.

Exp Hematol 2005 Sep;33(9):955-64

Department of Gene and Cell Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA.

View Article and Find Full Text PDF
September 2005

Restoration of NK T cell development in fyn-mutant mice by a TCR reveals a requirement for Fyn during early NK T cell ontogeny.

J Immunol 2004 May;172(10):6093-100

Graduate Group in Immunology, University of Pennsylvania, Philadelphia, PA 19104, USA.

View Article and Find Full Text PDF
May 2004

NK T cell precursors exhibit differential cytokine regulation and require Itk for efficient maturation.

J Immunol 2002 Sep;169(5):2397-406

Graduate Group in Immunology and Department of Dermatology, University of Pennsylvania, Philadelphia 19104, USA.

View Article and Find Full Text PDF
September 2002