Brian P Sorrentino

Brian P Sorrentino

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Brian P Sorrentino

Publications by authors named "Brian P Sorrentino"

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

N Engl J Med 2019 04;380(16):1525-1534

From the Departments of Bone Marrow Transplantation and Cellular Therapy (E.M., B.T., W.J., S.G.), Hematology (S.Z., Z.M., J.C., J.D., X.T., B.Y.R., M.J.W., B.P.S.), Therapeutics Production and Quality (T.L., M.M.M.), Immunology (H.A., B.Y.), Pharmaceutical Sciences (S.J.C.), Biostatistics (G.K., C.L.), and Infectious Diseases (G.M.), St. Jude Children's Research Hospital, Memphis, TN; the Allergy and Clinical Immunology Division, Hospital Nacional Edgardo Rebagliati Martins, Lima, Peru (J.C.A.B.); the Department of Pediatrics, Allergy-Immunology Division, Children's Hospital Los Angeles, Los Angeles (J.A.C.), and the Department of Pediatrics, Division of Pediatric Allergy-Immunology-Bone Marrow Transplantation, University of California, San Francisco (UCSF) Benioff Children's Hospital, San Francisco (J.R.L.-B., J.M.P., M.J.C.) - both in California; the Department of Pediatrics, Pediatric Allergy and Immunology, University of New Mexico, Albuquerque (E.D.); University of Oklahoma Health Sciences Center, Tulsa (J.T.L.); Departamento de Pediatria da Universidade de Taubaté, Conselho Nacional de Medicina, São Paulo (A.C.M.A.); Copperfield Childcare, Claremont, South Africa (H.W.); and the Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD (S.S.D.R., H.L.M.).

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http://www.nejm.org/doi/10.1056/NEJMoa1815408
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http://dx.doi.org/10.1056/NEJMoa1815408DOI Listing
April 2019

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

Exp Hematol 2018 09 30;65:29-33. Epub 2018 Jun 30.

Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.

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http://dx.doi.org/10.1016/j.exphem.2018.06.286DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6128752PMC
September 2018

Evaluating the Safety of Retroviral Vectors Based on Insertional Oncogene Activation and Blocked Differentiation in Cultured Thymocytes.

Mol Ther 2016 Jun 9;24(6):1090-1099. Epub 2016 Mar 9.

Division of Experimental Hematology, Department of Hematology, Memphis, Tennessee, USA. Electronic address:

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http://dx.doi.org/10.1038/mt.2016.55DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4923324PMC
June 2016

Abcg2-Labeled Cells Contribute to Different Cell Populations in the Embryonic and Adult Heart.

Stem Cells Dev 2016 Feb 7;25(3):277-84. Epub 2016 Jan 7.

1 Department of Medicine, Lillehei Heart Institute, University of Minnesota , Minneapolis, Minnesota.

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http://dx.doi.org/10.1089/scd.2015.0272DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4742971PMC
February 2016

Gene therapy studies in a canine model of X-linked severe combined immunodeficiency.

Hum Gene Ther Clin Dev 2015 Mar 24;26(1):50-6. Epub 2015 Feb 24.

1 Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania , Philadelphia, PA 19104.

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http://dx.doi.org/10.1089/humc.2015.004DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4442583PMC
March 2015

Quantitative shearing linear amplification polymerase chain reaction: an improved method for quantifying lentiviral vector insertion sites in transplanted hematopoietic cell systems.

Hum Gene Ther Methods 2015 Feb 5;26(1):4-12. Epub 2015 Feb 5.

1 Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital , Memphis, TN 38120.

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http://dx.doi.org/10.1089/hgtb.2014.122DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4337463PMC
February 2015

Downregulation of Prdm16 mRNA is a specific antileukemic mechanism during HOXB4-mediated HSC expansion in vivo.

Blood 2014 Sep 31;124(11):1737-47. Epub 2014 Jul 31.

Experimental Hematology Division, Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN;

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http://dx.doi.org/10.1182/blood-2013-10-534735DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4162106PMC
September 2014

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

J Exp Med 2014 Apr 31;211(4):701-13. Epub 2014 Mar 31.

Department of Hematology and 2 Department of Pathology, Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105.

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http://www.jem.org/lookup/doi/10.1084/jem.20122727
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http://dx.doi.org/10.1084/jem.20122727DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3978278PMC
April 2014

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

PLoS One 2013 23;8(4):e62333. Epub 2013 Apr 23.

Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, United States of America.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0062333PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3633865PMC
November 2013

Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors.

Hum Gene Ther Clin Dev 2013 Jun 20;24(2):77-85. Epub 2013 Jun 20.

Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1089/humc.2012.244DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4003467PMC
June 2013

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

Drug Metab Dispos 2013 Apr 8;41(4):923-31. Epub 2013 Jan 8.

Department of Pharmaceutical Sciences, St. Jude Children’s Research Hospital, Memphis, Tennessee 38105, USA.

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http://dx.doi.org/10.1124/dmd.112.050344DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3608452PMC
April 2013

Abcg2 expression marks tissue-specific stem cells in multiple organs in a mouse progeny tracking model.

Stem Cells 2012 Feb;30(2):210-21

Division of Experimental Hematology, Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.

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http://dx.doi.org/10.1002/stem.1002DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4711397PMC
February 2012

Antibody binding shift assay for rapid screening of drug interactions with the human ABCG2 multidrug transporter.

Eur J Pharm Sci 2012 Jan 17;45(1-2):101-9. Epub 2011 Nov 17.

Membrane Research Group of Hungarian Academy of Sciences, Department of Biophysics, Semmelweis University and National Blood Center, Diószegi u 64, H-1113 Budapest, Hungary.

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http://dx.doi.org/10.1016/j.ejps.2011.10.021DOI Listing
January 2012

Abcg2 labels multiple cell types in skeletal muscle and participates in muscle regeneration.

J Cell Biol 2011 Oct 26;195(1):147-63. Epub 2011 Sep 26.

Department of Molecular, Cellular, and Developmental Biology, University of Colorado, Boulder, CO 80309, USA.

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http://dx.doi.org/10.1083/jcb.201103159DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3187700PMC
October 2011

Functional interactions between Lmo2, the Arf tumor suppressor, and Notch1 in murine T-cell malignancies.

Blood 2011 May 22;117(20):5453-62. Epub 2011 Mar 22.

Division of Experimental Hematology, Department of Hematology, St Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1182/blood-2010-09-309831DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3109717PMC
May 2011

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

Blood 2010 Aug 14;116(5):711-9. Epub 2010 Apr 14.

Experimental Hematology Division, Department of Hematology, St Jude Children's Research Hospital, Memphis, TN 38105-3678, USA.

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http://dx.doi.org/10.1182/blood-2009-07-235341DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2918328PMC
August 2010

A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells.

Blood 2010 Aug 10;116(6):900-8. Epub 2010 May 10.

Division of Experimental Hematology, Department of Hematology, St Jude Children's Research Hospital, 262 Danny Thomas Pl., Memphis, TN 38105, USA.

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http://dx.doi.org/10.1182/blood-2009-10-250209DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2924228PMC
August 2010

Scl and stem cell quiescence.

Blood 2010 Jan;115(4):751-2

ST. JUDE CHILDREN'S RESEARCH HOSPITAL.

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http://dx.doi.org/10.1182/blood-2009-11-251264DOI Listing
January 2010

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

Proc Natl Acad Sci U S A 2009 Apr 3;106(16):6685-90. Epub 2009 Apr 3.

Department of Developmental Neurobiology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1073/pnas.0901596106DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2672506PMC
April 2009

Expression of mouse Abcg2 mRNA during hematopoiesis is regulated by alternative use of multiple leader exons and promoters.

J Biol Chem 2006 Oct 1;281(40):29625-32. Epub 2006 Aug 1.

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

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http://www.jbc.org/lookup/doi/10.1074/jbc.M606314200
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http://dx.doi.org/10.1074/jbc.M606314200DOI Listing
October 2006

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy.

Proc Natl Acad Sci U S A 2006 Aug 24;103(31):11730-5. Epub 2006 Jul 24.

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1073/pnas.0603635103DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1518804PMC
August 2006

Prospective isolation of murine hematopoietic stem cells by expression of an Abcg2/GFP allele.

Stem Cells 2006 Jun 16;24(6):1556-63. Epub 2006 Feb 16.

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

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http://doi.wiley.com/10.1634/stemcells.2005-0562
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http://dx.doi.org/10.1634/stemcells.2005-0562DOI Listing
June 2006

Genotoxicity of retroviral integration in hematopoietic cells.

Mol Ther 2006 Jun 19;13(6):1031-49. Epub 2006 Apr 19.

Division of Experimental Hematology, Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, TN 38103, USA.

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http://dx.doi.org/10.1016/j.ymthe.2006.03.001DOI Listing
June 2006

A limited role for p16Ink4a and p19Arf in the loss of hematopoietic stem cells during proliferative stress.

Blood 2005 Aug 3;106(3):827-32. Epub 2005 Feb 3.

Department of Hematology/Oncology, St Jude Children's Research Hospital, 332 N Lauderdale St, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1182/blood-2004-06-2242DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1895157PMC
August 2005

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

Blood 2005 Mar 16;105(6):2571-6. Epub 2004 Nov 16.

Division of Experimental Hematology, Department of Hematology/Oncology, St Jude Children's Research Hospital, 332 N Lauderdale St, Memphis, TN 38105, USA.

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http://www.bloodjournal.org/cgi/doi/10.1182/blood-2004-04-15
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http://dx.doi.org/10.1182/blood-2004-04-1566DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4757428PMC
March 2005

Function-dependent conformational changes of the ABCG2 multidrug transporter modify its interaction with a monoclonal antibody on the cell surface.

J Biol Chem 2005 Feb 22;280(6):4219-27. Epub 2004 Nov 22.

National Medical Center, Institute of Haematology and Immunology, Membrane Research Group and Hungarian Academy of Sciences, 1113 Budapest, Hungary.

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http://www.jbc.org/lookup/doi/10.1074/jbc.M411338200
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http://dx.doi.org/10.1074/jbc.M411338200DOI Listing
February 2005

Clinical strategies for expansion of haematopoietic stem cells.

Nat Rev Immunol 2004 Nov;4(11):878-88

St. Jude Children's Research Hospital, Department of Hematology/Oncology, Division of Experimental Hematology, 332 North Lauderdale, Memphis, Tennessee 38120, USA.

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http://www.nature.com/articles/nri1487
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http://dx.doi.org/10.1038/nri1487DOI Listing
November 2004

The stem cell marker Bcrp/ABCG2 enhances hypoxic cell survival through interactions with heme.

J Biol Chem 2004 Jun 24;279(23):24218-25. Epub 2004 Mar 24.

Department of Pharmaceutical Sciences, St. Jude Children's Research Hospital, Memphis, Tennessee 38105-2794, USA.

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http://dx.doi.org/10.1074/jbc.M313599200DOI Listing
June 2004

Transient in vivo selection of transduced peripheral blood cells using antifolate drug selection in rhesus macaques that received transplants with hematopoietic stem cells expressing dihydrofolate reductase vectors.

Blood 2004 Feb 14;103(3):796-803. Epub 2003 Aug 14.

Department of Hematology-Oncology, Division of Experimental Hematology, St. Jude Children's Research Hospital, 332 N Lauderdale, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1182/blood-2003-05-1572DOI Listing
February 2004

Hematopoietic cells from mice that are deficient in both Bcrp1/Abcg2 and Mdr1a/1b develop normally but are sensitized to mitoxantrone.

Biotechniques 2003 Dec;35(6):1248-52

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, 332 N. Lauderdale Street, Memphis, TN 38105, USA.

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https://www.future-science.com/doi/10.2144/03356ss04
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http://dx.doi.org/10.2144/03356ss04DOI Listing
December 2003

Reduction in hematopoietic stem cell numbers with in vivo drug selection can be partially abrogated by HOXB4 gene expression.

Mol Ther 2003 Sep;8(3):376-84

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, 332 North Lauderdale, 38105, Memphis, Tennessee 38105, USA.

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September 2003

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells.

Blood 2003 Jul 27;102(2):506-13. Epub 2003 Mar 27.

Division of Experimental Hematology, Department of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.

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http://dx.doi.org/10.1182/blood-2003-03-0677DOI Listing
July 2003

Development of gene therapy for hemoglobin disorders.

Ann N Y Acad Sci 2003 May;996:101-11

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

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May 2003

Low levels of ABCG2 expression in adult AML blast samples.

Blood 2002 Dec 25;100(13):4594-601. Epub 2002 Jul 25.

Department of Hematology/Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1182/blood-2002-01-0271DOI Listing
December 2002

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.

Proc Natl Acad Sci U S A 2002 Sep 6;99(19):12339-44. Epub 2002 Sep 6.

Division of Experimental Hematology, Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

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http://dx.doi.org/10.1073/pnas.192276999DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC129446PMC
September 2002

Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs.

Nat Rev Cancer 2002 Jun;2(6):431-41

Department of Hematology/Oncology, Division of Experimental Hematology, St Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.

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http://dx.doi.org/10.1038/nrc823DOI Listing
June 2002

Reduced lymphomyeloid repopulating activity from adult bone marrow and fetal liver of mice lacking expression of STAT5.

Blood 2002 Jan;99(2):479-87

Hematopoiesis Department, American Red Cross Holland Laboratory, Rockville, MD, USA.

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January 2002