Publications by authors named "Donald B Kohn"

98Publications

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry.

J Clin Immunol 2020 Sep 2. Epub 2020 Sep 2.

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, 90095, USA.

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http://dx.doi.org/10.1007/s10875-020-00857-9DOI Listing
September 2020

Overview of the current status of gene therapy for primary immune deficiencies (PIDs).

J Allergy Clin Immunol 2020 Aug;146(2):229-233

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Calif; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Calif; Department of Molecular & Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Calif; Department of Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, David Geffen School of Medicine, University of California, Los Angeles, Calif. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2020.05.024DOI Listing
August 2020

AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation.

Biol Blood Marrow Transplant 2020 Jul 28. Epub 2020 Jul 28.

Department of Pediatrics, Division of Pediatric Hematology-Oncology, University of California, Los Angeles, Los Angeles, California; Pediatric Blood and Marrow Transplant Program, University of California, Los Angeles, Los Angeles, California. Electronic address:

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http://dx.doi.org/10.1016/j.bbmt.2020.07.029DOI Listing
July 2020

Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.

Biol Blood Marrow Transplant 2020 Jul 9. Epub 2020 Jul 9.

Department of Pediatric Hematology/Oncology, David Geffen School of Medicine at UCLA, Los Angeles, California; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, California; The Broad Stem Cell Research Center, University of California, Los Angeles, California. Electronic address:

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http://dx.doi.org/10.1016/j.bbmt.2020.07.004DOI Listing
July 2020

Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences.

Mol Ther Methods Clin Dev 2020 Jun 18;17:999-1013. Epub 2020 Apr 18.

Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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http://dx.doi.org/10.1016/j.omtm.2020.04.006DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7225380PMC
June 2020

Dosing and Re-Administration of Lentiviral Vector for Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice.

Mol Ther Methods Clin Dev 2020 Mar 16;16:78-93. Epub 2019 Nov 16.

Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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http://dx.doi.org/10.1016/j.omtm.2019.11.004DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909201PMC
March 2020

Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements.

Mol Ther 2020 01 28;28(1):328-340. Epub 2019 Sep 28.

Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; The Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2019.09.020DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6953778PMC
January 2020

Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer.

Cell Stem Cell 2019 10 5;25(4):542-557.e9. Epub 2019 Sep 5.

Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, Los Angeles, CA 90095, USA; Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address:

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http://dx.doi.org/10.1016/j.stem.2019.08.004DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7018522PMC
October 2019

Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.

Mol Ther 2019 08 24;27(8):1389-1406. Epub 2019 May 24.

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA, USA; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2019.05.014DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6697408PMC
August 2019

PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector.

Mol Ther Methods Clin Dev 2019 Jun 4;13:390-398. Epub 2019 Apr 4.

Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA.

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http://dx.doi.org/10.1016/j.omtm.2019.03.005DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6477655PMC
June 2019

Chemistry, manufacturing and controls for gene modified hematopoietic stem cells.

Cytotherapy 2019 03 8;21(3):358-366. Epub 2019 Feb 8.

Department of Microbiology, Immunology and Molecular Genetics, University of California Los Angeles, Los Angeles, California, USA.

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http://dx.doi.org/10.1016/j.jcyt.2018.12.001DOI Listing
March 2019

Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome.

Cell Stem Cell 2019 02 10;24(2):309-317.e7. Epub 2019 Jan 10.

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address:

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https://linkinghub.elsevier.com/retrieve/pii/S19345909183059
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http://dx.doi.org/10.1016/j.stem.2018.12.003DOI Listing
February 2019

Gene therapy for blood diseases.

Authors:
Donald B Kohn

Curr Opin Biotechnol 2019 12 29;60:39-45. Epub 2018 Dec 29.

Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Pediatrics, University of California, Los Angeles, David Geffen School of Medicine, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, David Geffen School of Medicine, USA. Electronic address:

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http://dx.doi.org/10.1016/j.copbio.2018.11.016DOI Listing
December 2019

Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.

Hum Gene Ther 2018 10;29(10):1153-1166

1 Department of Microbiology, Immunology, and Molecular Genetics, University of California , Los Angeles, California; University of Modena and Reggio Emilia , Italy .

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https://www.liebertpub.com/doi/10.1089/hum.2018.061
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http://dx.doi.org/10.1089/hum.2018.061DOI Listing
October 2018

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

J Allergy Clin Immunol 2019 03 5;143(3):852-863. Epub 2018 Sep 5.

Division of Immunology and Allergy, and the Department of Pediatrics, Developmental and Stem Cell Biology Program, Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address:

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http://dx.doi.org/10.1016/j.jaci.2018.08.024DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6688493PMC
March 2019

Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization.

Gene Ther 2018 10 6;25(7):454-472. Epub 2018 Sep 6.

Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, 27599, USA.

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http://dx.doi.org/10.1038/s41434-018-0039-2DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6478381PMC
October 2018

SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.

Blood 2018 10 28;132(17):1737-1749. Epub 2018 Aug 28.

Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.

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http://dx.doi.org/10.1182/blood-2018-03-840702DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6202916PMC
October 2018

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome.

Cell Rep 2018 05;23(9):2606-2616

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Division of Hematology & Oncology, Department of Pediatrics, David Geffen School of Medicine at the University of California, Los Angeles, Los Angeles, CA 90095, USA.

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http://dx.doi.org/10.1016/j.celrep.2018.04.103DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6181643PMC
May 2018

Leukocyte adhesion deficiency-I: A comprehensive review of all published cases.

J Allergy Clin Immunol Pract 2018 Jul - Aug;6(4):1418-1420.e10. Epub 2018 Jan 20.

Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain; Advanced Therapies Unit, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), Madrid, Spain.

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http://dx.doi.org/10.1016/j.jaip.2017.12.008DOI Listing
November 2019

Gene therapy comes of age.

Science 2018 01;359(6372)

Memorial Sloan Kettering Cancer Center, New York, NY, USA.

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http://dx.doi.org/10.1126/science.aan4672DOI Listing
January 2018

Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.

Cell Stem Cell 2017 Nov;21(5):574-590

Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; The Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, CA, USA. Electronic address:

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http://dx.doi.org/10.1016/j.stem.2017.10.010DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6039108PMC
November 2017

Gene therapy: WAS (not) just for kids.

Authors:
Donald B Kohn

Blood 2017 09;130(11):1278-1279

UNIVERSITY OF CALIFORNIA, LOS ANGELES.

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http://dx.doi.org/10.1182/blood-2017-08-798496DOI Listing
September 2017

Gene Therapy.

Hematol Oncol Clin North Am 2017 10 7;31(5):xiii-xiv. Epub 2017 Aug 7.

Department of Microbiology, Department of Immunology & Molecular Genetics, Department of Pediatrics, Department of Molecular & Medical Pharmacology, UCLA Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, David Geffen School of Medicine, University of California, Los Angeles, 3163 Terasaki Life Science Building, 610 Charles E. Young Drive East, Los Angeles, CA 90095, USA. Electronic address:

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http://dx.doi.org/10.1016/j.hoc.2017.07.001DOI Listing
October 2017

Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy.

Authors:
Donald B Kohn

Hematol Oncol Clin North Am 2017 10;31(5):721-735

Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; Division of Hematology/Oncology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA, USA; UCLA Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address:

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http://dx.doi.org/10.1016/j.hoc.2017.06.006DOI Listing
October 2017

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations.

J Clin Immunol 2017 Oct 25;37(7):626-637. Epub 2017 Aug 25.

Department of Pediatrics, University of California, Los Angeles (UCLA), 3163 Terasaki Life Science Bldg., 610 Charles E. Young Drive East, Los Angeles, CA, 90095, USA.

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http://dx.doi.org/10.1007/s10875-017-0433-3DOI Listing
October 2017

Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.

Cytotherapy 2017 09 18;19(9):1096-1112. Epub 2017 Jul 18.

Department of Microbiology, Immunology and Molecular Genetics and the Eli & Edythe Broad Stem Cell Research Center, University of California, Los Angeles, California, USA. Electronic address:

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http://dx.doi.org/10.1016/j.jcyt.2017.06.002DOI Listing
September 2017

Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells.

Mol Ther 2017 09 27;25(9):2163-2175. Epub 2017 Jun 27.

Department of Microbiology, Immunology & Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Molecular & Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2017.05.023DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5589063PMC
September 2017

New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.

J Allergy Clin Immunol 2017 Mar;139(3):726-732

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Calif.

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http://dx.doi.org/10.1016/j.jaci.2017.01.007DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5911283PMC
March 2017

How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).

J Clin Immunol 2017 May 14;37(4):351-356. Epub 2017 Feb 14.

Molecular and Cellular Immunology Section, UCL Institute of Child Health, University College London, London, UK.

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http://dx.doi.org/10.1007/s10875-017-0373-yDOI Listing
May 2017

Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.

Mol Ther Nucleic Acids 2016 ;5:e351

Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA; Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA. Electronic address:

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http://dx.doi.org/10.1038/mtna.2016.52DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5023398PMC
January 2016

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.

Sci Transl Med 2016 10;8(360):360ra134

Innovative Genomics Initiative, University of California, Berkeley, Berkeley, CA 94720, USA. Department of Molecular and Cellular Biology, University of California, Berkeley, Berkeley, CA 94720, USA.

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http://stm.sciencemag.org/content/scitransmed/8/360/360ra134
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http://dx.doi.org/10.1126/scitranslmed.aaf9336DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5500303PMC
October 2016

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.

Hum Gene Ther 2017 01 7;28(1):112-124. Epub 2016 Sep 7.

1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.

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http://dx.doi.org/10.1089/hum.2016.064DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5278830PMC
January 2017

Analyzing CRISPR genome-editing experiments with CRISPResso.

Nat Biotechnol 2016 07;34(7):695-7

Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA.

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http://dx.doi.org/10.1038/nbt.3583DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5242601PMC
July 2016

Primary Immune Deficiency Treatment Consortium (PIDTC) update.

J Allergy Clin Immunol 2016 08 22;138(2):375-85. Epub 2016 Apr 22.

Pediatric Immunology and Pediatrics, Mother and Child Ste-Justine Hospital, University of Montreal, Montreal, Quebec, Canada.

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http://dx.doi.org/10.1016/j.jaci.2016.01.051DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4986691PMC
August 2016

Gene Therapy for the Treatment of Primary Immune Deficiencies.

Curr Allergy Asthma Rep 2016 May;16(5):39

Division of Hematology & Oncology, Department of Pediatrics, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.

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http://dx.doi.org/10.1007/s11882-016-0615-8DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5909980PMC
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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http://dx.doi.org/10.1182/blood-2016-01-678136DOI Listing
May 2016

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells.

Cell Stem Cell 2016 Apr 11;18(4):533-40. Epub 2016 Feb 11.

Molecular Biology Institute, University of California, Los Angeles, CA 90095, USA; Center for Duchenne Muscular Dystrophy, University of California, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, University of California, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, CA 90095, USA. Electronic address:

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http://www.cell.com/cell-stem-cell/pdf/S1934-5909(16)00022-9
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http://linkinghub.elsevier.com/retrieve/pii/S193459091600022
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http://dx.doi.org/10.1016/j.stem.2016.01.021DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4826286PMC
April 2016

Delivery of Genome Editing Reagents to Hematopoietic Stem/Progenitor Cells.

Curr Protoc Stem Cell Biol 2016 Feb 3;36:5B.4.1-5B.4.10. Epub 2016 Feb 3.

Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California.

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http://dx.doi.org/10.1002/9780470151808.sc05b04s36DOI Listing
February 2016

Impulse oscillometry identifies peripheral airway dysfunction in children with adenosine deaminase deficiency.

Orphanet J Rare Dis 2015 Dec 18;10:159. Epub 2015 Dec 18.

National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, 20892, USA.

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http://dx.doi.org/10.1186/s13023-015-0365-zDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4683718PMC
December 2015

Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection.

Am J Hematol 2015 Nov 12;90(11):1021-6. Epub 2015 Oct 12.

Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Hospital Los Angeles, University of Southern California, Los Angeles, California.

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http://dx.doi.org/10.1002/ajh.24141DOI Listing
November 2015

Gene therapy outpaces haplo for SCID-X1.

Authors:
Donald B Kohn

Blood 2015 Jun;125(23):3521-2

UNIVERSITY OF CALIFORNIA, LOS ANGELES.

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http://dx.doi.org/10.1182/blood-2015-04-641720DOI Listing
June 2015

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells.

Mol Ther Methods Clin Dev 2015 22;2:15012. Epub 2015 Apr 22.

Department of Microbiology, Immunology and Molecular Genetics, University of California , Los Angeles, California, USA ; Department of Pediatrics, UCLA Children's Discovery and Innovation Institute David Geffen School of Medicine, University of California , Los Angeles, California, USA.

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https://linkinghub.elsevier.com/retrieve/pii/S23290501163002
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http://dx.doi.org/10.1038/mtm.2015.12DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445009PMC
June 2015

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.

Blood 2015 Apr 2;125(17):2597-604. Epub 2015 Mar 2.

Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA.

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http://dx.doi.org/10.1182/blood-2014-12-615948DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4408287PMC
April 2015

Eliminating SCID row: new approaches to SCID.

Authors:
Donald B Kohn

Hematology Am Soc Hematol Educ Program 2014 Dec 18;2014(1):475-80. Epub 2014 Nov 18.

Departments of Microbiology, Immunology, and Molecular Genetics and Pediatrics; David Geffen School of Medicine, Mattel Children's Hospital; and Eli & Edythe Broad Center for Regenerative Medicine and Stem Cells, University of California, Los Angeles, CA.

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http://dx.doi.org/10.1182/asheducation-2014.1.475DOI Listing
December 2014

Putative immunogenicity expression profiling using human pluripotent stem cells and derivatives.

Stem Cells Transl Med 2015 Feb 9;4(2):136-45. Epub 2015 Jan 9.

Department of Molecular and Medical Pharmacology, Department of Microbiology, Immunology, and Molecular Genetics, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, Department of Pediatrics, Mattel Children's Hospital, Jonsson Comprehensive Cancer Center, and Department of Medicine, UCLA School of Medicine, University of California, Los Angeles, Los Angeles, California, USA

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http://dx.doi.org/10.5966/sctm.2014-0117DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4303355PMC
February 2015

Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter.

Nucleic Acids Res 2015 Jan 17;43(1):682-90. Epub 2014 Dec 17.

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA 90095, USA Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA 90095, USA Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA

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http://dx.doi.org/10.1093/nar/gku1312DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4288199PMC
January 2015

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

N Engl J Med 2014 Oct;371(15):1407-17

From the Departments of Biotherapy (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.) and Immunology and Pediatric Hematology (S.B., G.C., D.M., B.N., C.P., F.T., A.F.) and the Centre d'Étude des Déficits Immunitaires (C.P.), Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), the Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AP-HP, INSERM (S.H.-B.-A., J. Blondeau, L.C., F.T., M.C.), Unité de Technologies Chimiques et Biologiques pour la Santé, Centre National de la Recherche Scientifique, 8258-INSERM Unité 1022, Faculté des Sciences Pharmaceutiques et Biologiques, Université Paris Descartes (S.H.-B.-A.), Immunology Laboratory, Groupe Hospitalier Universitaire Paris-Sud, AP-HP, Le Kremlin-Bicêtre (S.H.-B.-A.), Imagine Institute, Paris Descartes-Sorbonne Paris Cité University (S.B., J. Blondeau, L.C., D.M., B.N., C.P., E.S., A.F., M.C.), INSERM Unités Mixtes de Recherche 1163, Laboratory of Human Lymphohematopoiesis (J. Blondeau, L.C., E.S., F.T., A.F., M.C.), Groupe Immunoscope, Immunology Department, Institut Pasteur (A.L.), and Collège de France (A.F.) - all in Paris; Division of Hematology-Oncology (S.-Y.P., H.B., D.G., C.E.H., G.H., L.E.L., W.B.L., D.A.W.) and Division of Immunology (L.D.N.), Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute (S.-Y.P., D.G., L.E.L., W.B.L., D.A.W.), Harvard Medical School (S.-Y.P., M.A., L.E.L., W.B.L., J.R., L.E.S., A.T., L.D.N., D.A.W.), Center for Human Cell Therapy, Program in Cellular and Molecular Medicine, Boston Children's Hospital (M.A., J.R., L.E.S., A.T.), Division of Hematologic Malignancies, Dana-Farber Cancer Institute (J.R.), and the Manton Center for Orphan Disease Research (L.D.N.) - all in Boston; Great Ormond Street Hospital for Children NHS Foundation Trust (H.B.G., J.X.-B., A.J.T.) and Section of Molecular and Cellular Immunology, University College London Institute of Child Health (H.B.G., K.F.B., A.

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http://dx.doi.org/10.1056/NEJMoa1404588DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4274995PMC
October 2014

Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States.

JAMA 2014 Aug;312(7):729-38

Department of Pediatrics, University of California, San Francisco, San Francisco2UCSF Benioff Children's Hospital, San Francisco, California.

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http://primaryimmune.org/wp-content/uploads/2014/10/August-2
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http://jama.jamanetwork.com/article.aspx?doi=10.1001/jama.20
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http://dx.doi.org/10.1001/jama.2014.9132DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4492158PMC
August 2014

Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.

Mol Ther 2014 Oct 13;22(10):1803-16. Epub 2014 Jun 13.

1] Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, California, USA [2] Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, California, USA.

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http://dx.doi.org/10.1038/mt.2014.88DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4428412PMC
October 2014

Human lymphoid development in the absence of common γ-chain receptor signaling.

J Immunol 2014 Jun 25;192(11):5050-8. Epub 2014 Apr 25.

Department of Pathology and Laboratory Medicine, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles CA 90095;

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http://dx.doi.org/10.4049/jimmunol.1303496DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4052377PMC
June 2014

HIV eradication--from Berlin to Boston.

Nat Biotechnol 2014 Apr;32(4):315-6

Fred Hutchinson Cancer Research Center and University of Washington, Seattle, Washington, USA.

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http://dx.doi.org/10.1038/nbt.2868DOI Listing
April 2014

Adoptive transfer of MART-1 T-cell receptor transgenic lymphocytes and dendritic cell vaccination in patients with metastatic melanoma.

Clin Cancer Res 2014 May 14;20(9):2457-65. Epub 2014 Mar 14.

Authors' Affiliations: Departments of Medicine, Surgery, Pathology and Laboratory Medicine, Microbiology, Immunology and Molecular Genetics, and Molecular and Medical Pharmacology; Jonsson Comprehensive Cancer Center; Department of Ophthalmology, Jules Stein Eye Institute; Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research; Howard Hughes Medical Institute, University of California, Los Angeles (UCLA); The Angeles Clinic Research Institute, Los Angeles; Department of Medicine, University of California San Diego (UCSD) Moores Cancer Center, La Jolla; Divisions of Chemistry and Biology, California Institute of Technology, Pasadena, California; Department of Medical and Molecular Genetics, Indiana University, and the Indiana University Viral Production Facility (IU VPF), Indianapolis, Indiana; Comprehensive Cancer Centers of Nevada, Las Vegas, Nevada; Mayo Clinic Scottsdale, Scottsdale, Arizona; Department of Medicine, University of Connecticut Health Center, Farmington, Connecticut; and Center for Immunology, Roswell Park Cancer Institute, Buffalo, New York.

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http://clincancerres.aacrjournals.org/cgi/doi/10.1158/1078-0
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http://dx.doi.org/10.1158/1078-0432.CCR-13-3017DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4070853PMC
May 2014

Dissecting the mechanism of histone deacetylase inhibitors to enhance the activity of zinc finger nucleases delivered by integrase-defective lentiviral vectors.

Hum Gene Ther 2014 Jul 2;25(7):599-608. Epub 2014 Apr 2.

Department of Microbiology, Immunology, and Molecular Genetics; and Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research; University of California , Los Angeles, Los Angeles, CA 90095.

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http://dx.doi.org/10.1089/hum.2013.211DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4098962PMC
July 2014

Hematopoietic stem cells for cancer immunotherapy.

Immunol Rev 2014 Jan;257(1):237-49

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, David Geffen School of Medicine, Los Angeles, CA, USA; Eli and Edythe Broad Center for Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, David Geffen School of Medicine, Los Angeles, CA, USA.

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http://dx.doi.org/10.1111/imr.12128DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3901057PMC
January 2014

Prostate cancer originating in basal cells progresses to adenocarcinoma propagated by luminal-like cells.

Proc Natl Acad Sci U S A 2013 Dec 26;110(50):20111-6. Epub 2013 Nov 26.

Microbiology, Immunology and Molecular Genetics, Molecular Biology Interdepartmental Ph.D. Program, Departments of Molecular and Medical Pharmacology, Pathology and Laboratory Medicine, Jonsson Comprehensive Cancer Center, David Geffen School of Medicine, Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, Howard Hughes Medical Institute, and Department of Urology, University of California, Los Angeles, CA 90095.

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http://dx.doi.org/10.1073/pnas.1320565110DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3864278PMC
December 2013

Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus.

Mol Ther 2013 Sep 16;21(9):1705-17. Epub 2013 Jul 16.

Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, California 90095, USA

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http://dx.doi.org/10.1038/mt.2013.106DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3776625PMC
September 2013