Publications by authors named "Matthew H Porteus"

93Publications

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

Nat Commun 2021 01 20;12(1):472. Epub 2021 Jan 20.

Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, 94305, USA.

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January 2021

The Binns Program for Cord Blood Research: A novel model of cord blood banking for academic biomedical research.

Placenta 2020 Oct 13;103:50-52. Epub 2020 Oct 13.

Division of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, 265 Campus Drive, Stanford, CA, 94305, USA. Electronic address:

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October 2020

Loss of Extreme Long-Range Enhancers in Human Neural Crest Drives a Craniofacial Disorder.

Cell Stem Cell 2020 Nov 28;27(5):765-783.e14. Epub 2020 Sep 28.

Department of Chemical and Systems Biology, Stanford University School of Medicine, Stanford, CA 94305, USA; Department of Developmental Biology, Stanford University School of Medicine, Stanford, CA 94305, USA; Institute of Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA; Howard Hughes Medical Institute, Stanford University School of Medicine, Stanford, CA 94305, USA. Electronic address:

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November 2020

Metabolic engineering generates a transgene-free safety switch for cell therapy.

Nat Biotechnol 2020 12 13;38(12):1441-1450. Epub 2020 Jul 13.

Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.

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December 2020

Macrophage Subpopulation Dynamics Shift following Intravenous Infusion of Mesenchymal Stromal Cells.

Mol Ther 2020 09 30;28(9):2007-2022. Epub 2020 May 30.

Hagey Laboratory for Pediatric Regenerative Medicine, Division of Plastic and Reconstructive Surgery, Department of Surgery, Stanford University School of Medicine, Stanford, CA, USA; Stanford Diabetes Research Center, Stanford, CA 95305, USA. Electronic address:

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September 2020

Improving the safety of human pluripotent stem cell therapies using genome-edited orthogonal safeguards.

Nat Commun 2020 06 1;11(1):2713. Epub 2020 Jun 1.

Institute for Stem Cell Biology & Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, 94305, USA.

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June 2020

Reply to "Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors".

Mol Ther 2019 12 11;27(12):2063. Epub 2019 Nov 11.

Department of Pediatrics, Stanford, University, Stanford, CA 94305, USA. Electronic address:

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December 2019

AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs.

Mol Ther 2019 10 12;27(10):1701-1705. Epub 2019 Sep 12.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address:

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October 2019

A New Class of Medicines through DNA Editing.

N Engl J Med 2019 03;380(10):947-959

From the Department of Pediatrics-Stem Cell Transplantation, Stanford University, Stanford, CA.

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March 2019

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements.

Mol Ther 2019 01 17;27(1):137-150. Epub 2018 Oct 17.

Genethon, INSERM UMR951, Evry 91000, France; Laboratory of Chromatin and Gene Regulation During Development, Imagine Institute, INSERM UMR1163, Paris 75015, France; Paris Descartes, Sorbonne Paris Cité University, Imagine Institute, Paris 75015, France. Electronic address:

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January 2019

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

Mol Ther Nucleic Acids 2018 Sep 3;12:89-104. Epub 2018 May 3.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address:

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September 2018

Gene Editing on Center Stage.

Trends Genet 2018 08 13;34(8):600-611. Epub 2018 Jun 13.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address:

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August 2018

CRISPR/Cas9 genome editing in human hematopoietic stem cells.

Nat Protoc 2018 02 25;13(2):358-376. Epub 2018 Jan 25.

Department of Pediatrics, Stanford University, Stanford, California, USA.

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February 2018

Genome Editing for the β-Hemoglobinopathies.

Adv Exp Med Biol 2017 ;1013:203-217

Department of Pediatrics, Stanford University, Lorry Lokey Stem Cell Research Building MC5462, 1291 Welch Rd, Stanford, CA, 94305, USA.

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April 2018

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

Curr Opin Hematol 2017 Nov;24(6):481-488

Department of Pediatrics, Stanford University, Stanford, California, USA.

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November 2017

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.

Cell Rep 2017 07;20(3):750-756

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address:

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July 2017

A Comprehensive TALEN-Based Knockout Library for Generating Human-Induced Pluripotent Stem Cell-Based Models for Cardiovascular Diseases.

Circ Res 2017 May 28;120(10):1561-1571. Epub 2017 Feb 28.

From the Stanford Cardiovascular Institute (I.K., V.T., J.L., S.D., I.I., M.A., R.S., H.W., N.M., N.-Y.S., T.S., N.W., K.D.W., E.M., J.C.W.), Department of Cardiothoracic Surgery (I.K.), Division of Cardiovascular Medicine, Department of Medicine (V.T., J.C.W.), CA; Institute of Stem Cell Biology and Regenerative Medicine (D.A.C., V.S., J.C.W.), Departments of Pediatrics (A.H., M.H.P.), Pathology (K.D.W.), and Obstetrics and Gynecology (V.S.), Stanford University School of Medicine, CA; Berlin Institute of Health, Germany (S.D.); and Max Delbrueck Center, Berlin, Germany (S.D.).

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May 2017

Knock-in editing: it functionally corrects!

Blood 2016 05;127(21):2507-9

STANFORD UNIVERSITY.

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May 2016

Ethical and regulatory aspects of genome editing.

Blood 2016 05 6;127(21):2553-60. Epub 2016 Apr 6.

Departments of Pediatrics and Immunology, University of Washington School of Medicine, Seattle, WA; and Program in Cell and Gene Therapy, Seattle Children's Research Institute, Seattle, WA.

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May 2016

Towards a new era in medicine: therapeutic genome editing.

Genome Biol 2015 Dec 22;16:286. Epub 2015 Dec 22.

Department of Pediatrics, Stanford University, Welch Road, Stanford, CA, 94305, USA.

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December 2015

Use of Genome Engineering to Create Patient Specific MLL Translocations in Primary Human Hematopoietic Stem and Progenitor Cells.

PLoS One 2015 9;10(9):e0136644. Epub 2015 Sep 9.

Division of Pediatric Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University, Stanford, California, United States of America.

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May 2016

Genome Editing of the Blood: Opportunities and Challenges.

Curr Stem Cell Rep 2015 Mar;1(1):23-30

Dept. of Pediatrics MC5462, Stanford University, Stanford, CA 94305,

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March 2015

Genome editing of the germline: broadening the discussion.

Mol Ther 2015 Jun;23(6):980-982

Department of Chemistry, Indiana University, Bloomington, Indiana, USA. Electronic address:

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June 2015

Quantifying on- and off-target genome editing.

Trends Biotechnol 2015 Feb 13;33(2):132-40. Epub 2015 Jan 13.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA. Electronic address:

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February 2015

Genome editing in mouse spermatogonial stem/progenitor cells using engineered nucleases.

PLoS One 2014 19;9(11):e112652. Epub 2014 Nov 19.

Department of Chemistry, Indiana University, Bloomington, Indiana, United States of America.

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July 2015

Gene/cell therapy approaches for Immune Dysregulation Polyendocrinopathy Enteropathy X-linked syndrome.

Curr Gene Ther 2014 ;14(6):422-8

San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Via Olgettina 58, 20131, Milan, Italy.

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July 2015

Genome editing of mouse fibroblasts by homologous recombination for sustained secretion of PDGF-B and augmentation of wound healing.

Plast Reconstr Surg 2014 Sep;134(3):389e-401e

Dallas, Texas; and Stanford, Calif. From the Department of Plastic Surgery, the Medical Scientist Training Program, the Animal Resource Center, Veterinary Pathology, and the Department of Cell Biology, The University of Texas Southwestern Medical Center; the Department of Surgery, Baylor University Medical Center; and the Department of Pediatrics, Stanford University.

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September 2014

Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing.

Cell Rep 2014 Apr 27;7(1):293-305. Epub 2014 Mar 27.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.

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April 2014

SAPTA: a new design tool for improving TALE nuclease activity.

Nucleic Acids Res 2014 Apr 16;42(6):e47. Epub 2014 Jan 16.

Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA, 30332, USA and Department of Pediatrics, Stanford University, Stanford, CA, 94305, USA.

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April 2014

Nuclease-mediated gene editing by homologous recombination of the human globin locus.

Nucleic Acids Res 2014 Jan 23;42(2):1365-78. Epub 2013 Oct 23.

Department of Pediatrics, Stanford University, 1291 Welch Rd. Stanford, CA 94305, USA and Department of Pediatrics, University of Texas Southwestern Medical Center, 5323 Harry Hines Blvd. Dallas, TX 75390, USA.

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January 2014

Receptor-mediated delivery of engineered nucleases for genome modification.

Nucleic Acids Res 2013 Oct 16;41(19):e182. Epub 2013 Aug 16.

Department of Anesthesiology and Perioperative Medicine, Georgia Regents University, 1120 15th Street, Augusta, GA 30912, USA, Department of Radiation Oncology, Emory University School of Medicine, 4121 Rollins Research Center, 1510 Clifton Rd. NE, Atlanta, GA 30322, USA, Cold Spring Harbor Laboratory, 1 Bungtown Road, Cold Spring Harbor, New York 11724, USA, Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA 30322, USA, Department of Pediatrics-Divisions of Hematology/Oncology and Human Gene Therapy, Stanford University School of Medicine, Stanford, CA 94305, USA and Department of Biochemistry, Emory University School of Medicine, 4121 Rollins Research Center, 1510 Clifton Rd. NE, Atlanta, GA 30322, USA.

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October 2013

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification.

Mol Ther Nucleic Acids 2013 Apr 30;2:e88. Epub 2013 Apr 30.

1] Department of Biochemistry, University of Texas Southwestern Medical Center, Dallas, Texas, USA [2] Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.

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April 2013

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus.

Mol Ther Nucleic Acids 2013 Apr 23;2:e87. Epub 2013 Apr 23.

1] Department of Biochemistry, University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, USA [2] Department of Pediatrics, University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, USA.

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April 2013

A crisper look at genome editing: RNA-guided genome modification.

Mol Ther 2013 Apr;21(4):720-2

Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

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April 2013

Generation of an HIV resistant T-cell line by targeted "stacking" of restriction factors.

Mol Ther 2013 Apr 29;21(4):786-95. Epub 2013 Jan 29.

Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.

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April 2013

Development of nuclease-mediated site-specific genome modification.

Curr Opin Immunol 2012 Oct 13;24(5):609-16. Epub 2012 Sep 13.

Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.

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October 2012

Viral single-strand DNA induces p53-dependent apoptosis in human embryonic stem cells.

PLoS One 2011 17;6(11):e27520. Epub 2011 Nov 17.

Gene Therapy Center, University of North Carolina, Chapel Hill, North Carolina, United States of America.

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May 2012

Creating higher titer lentivirus with caffeine.

Hum Gene Ther 2011 Jan 12;22(1):93-100. Epub 2010 Dec 12.

Department of Biochemistry, University of Texas Southwestern Medical Center, Dallas, TX 75390-9148, USA.

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January 2011

Gene correction by homologous recombination with zinc finger nucleases in primary cells from a mouse model of a generic recessive genetic disease.

Mol Ther 2010 Jun 13;18(6):1103-10. Epub 2010 Apr 13.

Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.

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June 2010

Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels.

PLoS Genet 2009 Feb 13;5(2):e1000376. Epub 2009 Feb 13.

Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, United States of America.

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February 2009

Spermatogonial stem cell self-renewal requires OCT4, a factor downregulated during retinoic acid-induced differentiation.

Stem Cells 2008 Nov 21;26(11):2928-37. Epub 2008 Aug 21.

Departments of Pediatrics, University of Texas Southwestern Medical Center at Dallas, Dallas, Texas, USA.

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November 2008

Comparison of zinc finger nucleases for use in gene targeting in mammalian cells.

Mol Ther 2008 Apr 4;16(4):707-17. Epub 2008 Mar 4.

Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA.

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April 2008

Human SMC5/6 complex promotes sister chromatid homologous recombination by recruiting the SMC1/3 cohesin complex to double-strand breaks.

EMBO J 2006 Jul 29;25(14):3377-88. Epub 2006 Jun 29.

Department of Pharmacology, The University of Texas Southwestern Medical Center, Dallas, TX 75390-9041, USA.

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July 2006

Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells.

Nucleic Acids Res 2005 26;33(18):5978-90. Epub 2005 Oct 26.

Department of Environmental Health Sciences, The Johns Hopkins University Bloomberg School of Public Health, 615 North Wolfe Street, Baltimore, MD 21205-2179, USA.

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November 2005

Mammalian gene targeting with designed zinc finger nucleases.

Mol Ther 2006 Feb 19;13(2):438-46. Epub 2005 Sep 19.

Department of Pediatrics, University of Texas Southwestern Medical School, Dallas, 75390-9063, USA.

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February 2006

Gene targeting using zinc finger nucleases.

Nat Biotechnol 2005 Aug;23(8):967-73

Department of Pediatrics, University of Texas Southwestern Medical Center, USA.

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August 2005

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

Nature 2005 Jun 3;435(7042):646-51. Epub 2005 Apr 3.

Sangamo BioSciences, Inc., Pt. Richmond Tech Center 501, Canal Blvd, Suite A100 Richmond, California 94804, USA.

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June 2005

Chimeric nucleases stimulate gene targeting in human cells.

Science 2003 May;300(5620):763

Department of Biology, California Institute of Technology, Pasadena CA 91125, USA.

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May 2003