Brian P. Sorrentino, MD
Brian P. Sorrentino, MD

Brian P. Sorrentino, MD

Member, St. Jude Faculty

  • Director, Experimental Hematology Division
  • Director, Vector Production Facility




BS – Union College Schenectady, New York (Cum Laude)
MD – SUNY Upstate Medical Center College, Syracuse, New York (Cum Laude)

Research Interests

  • Gene therapy for blood and immune cell disorders, including severe combined immunodeficiency (SCID)
  • Lentiviral vector design and development to target hematopoietic stem cells
  • Hematopoietic stem cell self-renewal and leukemogenesis
  • Role of the Abcg2 transporter in stem cell biology

Selected Publications

De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL. Lentiviral hematopoietic stem cell gene therapy of X-linked severe combined immunodeficiency. Sci Transl Med 8:335-335ra57, 2016.

Zhou S, Fatima S, Ma Z, Wang YD, Lu T, Janke LJ, Du Y, Sorrentino BP. Evaluating the safety of retroviral vectors based on insertional oncogene activation and blocked differentiation in cultured thymocytes. Mol Ther March 9, 2016.

Doyle MJ, Maher TJ, Li Q, Garry M, Sorrentino BP, Martin CM. Abvg2-labeled cells contribute to different cell populations in the embryonic and adult heart. Stem Cells Dev 25(3):277-284, 2016.

Morfouace M, Cheepala S, Jackson S, Fukuda Y, Patel YT, Fatima S, Kawauchi D, Shelat AA, Stewart CF, Sorrentino BP, Schuetz JD, Roussel MF. ABCG2 Transporter Expression Impacts Group 3 Medulloblastoma Response to Chemotherapy. Cancer Res 75(18):3879-3889, 2015.

Felsburg PJ, De Ravin SS, Malech HL, Sorrentino BP, Burtner C, Kiem HP. Gene Therapy Studies in a Canine Model of X-Linked Severe Combined Immunodeficiency. Hum Gene Ther Clin Dev 26(1):50-56, 2015.

Zhou S, Bonner MA, Wang YD, Rapp S, De Ravin SS, Malech HL, Sorrentino BP. 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 26(1):4-12. 2015.

Yu H, Neale G, Zhang H, Lee HM, Ma Z, Zhou S, Forget BG, Sorrentino BP. Downregulation of Prdm16 mRNA is a specific antileukemic mechanism during HOXB4-mediated HSC expansion in vivo. Blood 124(11):1737-1747, 2014.

Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR. Charting a clear path: the ASGCT Standardized Pathways Conference. Mol Ther 22(7):1235-1238, 2014.

Maher TJ, Ren Y, Li Q, Braunlin E, Garry MG, Sorrentino BP, Martin CM. ATP-binding cassette transporter Abcg2 lineage contributes to the cardiac vasculature after oxidative stress. Am J Physiol Heart Circ Physiol 306(12):H1610-H1618, 2014.

Treanor LM, Zhou S, Janke L, Churchman ML, Ma Z, Lu T, Chen SC, Mullighan CG, Sorrentino BP. Interleukin-7 receptor mutants initiate early T cell precursor leukemia in murine thymocyte progenitors with multipotent potential. J Exp Med 211(4):701-713, 2014.

De Ravin SS, Gray JT, Throm RE, Spindler J, Kearney M, Wu X, Coffin JM, Hughes SH, Malderelli F, Sorrentino BP, Malech HL. False-positive HIV PCR test following ex vivo lentiviral gene transfer treatment of X-linked severe combined immunodeficiency vector. Mol Ther 22(2):244-245, 2014.

Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW. Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors. Hum Gene Ther Clin Dev 24(2):77-85, 2013.

Yasuda K, Cline C, Vogel P, Onciu M, Fatima S, Sorrentino BP, Thirumaran RK, Ekins S, Urade Y, Fujimori K, Schuetz EG. Drug transporters on arachnoid barrier cells contribute to the blood-cerebrospinal fluid barrier. Drug Metab Dispos 41(4):923-931, 2013.

O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Hum Gene Ther 24(4):355-362, 2013.

Zhou S, Ma Z, Lu T, Janke L, Gray JT, Sorrentino BP. Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vector. PLoS One 8(4):e62333, 2013.

Greene MR, Lockey T, Mehta PK, Kim YS, Eldridge PW, Gray JT, Sorrentino BP. Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line. Hum Gene Ther Methods 23(5):297-308, 2012.

Corrigan-Curay J, Cohen-Haguenauer O, O'Reilly M, Ross SR, Fan H, Rosenberg N, Somia N, King N, Friedmann T, Dunbar C, Aiuti A, Naldini L, Baum C, von Kalle C, Kiem HP, Montini E, Bushman F, Sorrentino BP, Carrondo M, Malech H, Gahrton G, Shapiro R, Wolff L, Rosenthal E, Jambou R, Zaia J, Kohn DB. Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy. Mol Ther 20(6):1084-1094, 2012.

Fatima S, Zhou S, Sorrentino BP. Abcg2 expression marks tissue-specific stem cells in multiple organs in a mouse progeny tracking model. Stem Cells 30(2):210-221, 2011.

Telbisz A, Hegedus C, Ozvegy-Laczka C, Goda K, Varady G, Takats Z, Szabo E, Sorrentino BP, Varadi A, Sarkadi B. Antibody binding shift assay for rapid screening of drug interactions with the human ABCG2 multidrug transporter. Eur J Pharm Sci 45(1-2):101-109, 2012.

Doyle MJ, Zhou S, Tanaka KK, Pisconti A, Farina NH, Sorrentino BP, Olwin BB. Abcg2 labels multiple cell types in skeletal muscle and participates in muscle regeneration. J Cell Biol 195(1):147-163, 2011.

Campbell PK, Zong Y, Yang S, Zhou S, Rubnitz JE, Sorrentino BP. Identification of a novel, tissue-specific ABCG2 promoter expressed in pediatric acute megakaryoblastic leukemia. Leuk Res 35(10):1321-1329, 2011.

Zhou S, Mody D, Deravin SS, Hauer J, Lu T, Ma Z, Hacein-Bey Abina S, Gray JT, Greene MR, Cavazzana-Calvo M, Malech HL, Sorrentino BP. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood 116(6):900-908, 2010.

Jiang J, Yu H, Shou Y, Neale G, Zhou S, Lu T, Sorrentino BP. Hemgn is a direct transcriptional target of HOXB4 and induces expansion of murine myeloid progenitor cells. Blood 116(5):711-719, 2010.

Treanor LM, Volanakis EJ, Zhou S, Lu T, Sherr CJ, Sorrentino BP. Functional interactions between Lmo2, the Arf tumor suppressor, and Notch1 in murine T-cell malignancies. Blood 117(20):5453-5462, 2011.

Sorrentino B. Assessing the risk of T-cell malignancies in mouse models of SCID-X1. Mol Ther 18(5):868-870, 2010.

Show More

Larochelle A, Choi U, Shou Y, Naumann N, Loktionova NA, Clevenger J, Krouse A, Metzger M, Donahue RE, Kang E, Stewart C, Persons DA, Malech H, Dunbar CE, Sorrentino BP. In vivo drug selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques. J Clin Invest 119(7):1952-63, 2009.

Throm RE, Ouma AA, Zhou S, Chandrasekaran A, Lockey T, Greene M, De Ravin SS, Moayeri M, Malech HL, Sorrentino BP, Gray JT. Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood 113:5104-10, 2009.

Cicero SA, Johnson D, Reynthens S, Frase S, Connell S, Chow LM, Baker SJ, Sorrentino BP, Dyer MA. Cells previously identified as retinal stem cells are pigmented ciliary epithelial cells. Proc Natl Acad Sci USA 106:6685-90, 2009.

Ozvegy-Laczka C, Laczkó R, Hegedus C, Litman T, Várady G, Goda K, Hegedus T, Dokholyan NV,Sorrentino BP, Váradi A, Sarkadi B. Interaction with the 5D3 monoclonal antibody is regulated by intramolecular rearrangements but not by covalent dimer formation of the human ABCG2 multidrug transporter. J Biol Chem 283:26059-70, 2008.

Will E, Bailey J, Schuesler T, Modlich U, Balcik B, Burzynski B, Witte D, Layh-Schmitt G, Rudolph C, Schlegelberger B, von Kalle C, Baum C, Sorrentino BP et al. Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials. Mol Ther 15:782-91, 2007.

Husain SM, Shou Y, Sorrentino BP, Handgretinger R. Isolation, molecular cloning and in vitro expression of rhesus monkey (Macaca mulatta) prominin-1.s1 complementary DNA encoding a potential hematopoietic stem cell antigen. Tissue Antigens 68:317-24. 2006.

Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, Otsu M, Sorrentino B, Scobie L, et al. Gene therapy, X-SCID transgene leukaemogenicity (letter to editor). Nature 443:7109, 2006.

Zong Y, Zhou S, Fatima S, Sorrentino BP. Expression of mouse Abcg2 mRNA during hematopoiesis is regulated by alternative use of multiple leader exons and promoters. J Biol Chem 281:29625-32, 2006.

Shou Y, Ma Z, Lu T, Sorrentino BP. Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proc Natl Acad Sci USA 103:11730-5, 2006.

Leggas M, Panetta JC, Zhuang Y, Schuetz JD, Johnston B, Bai F, Sorrentino B, Zhou S, Houghton PJ, Stewart CF. Gefitinib modulates the function of multiple ATP-binding cassette transporters in vivo.Cancer Research 66:4802-7, 2006.

Tadjali M, Zhou S, Rehg J, Sorrentino BP. Prospective isolation of murine hematopoietic stem cells by expression of an Abcg2/GFP allele. Stem Cells 24:1556-63, 2006.

Seggewiss R, Pittaluga S, Adler RL, Guenaga FJ, Ferguson C, Pilz I, Ryu B, Sorrentino BP, Young WS, et al. Acute myeloid leukemia associated with retroviral gene transfer to hematopoietic progenitor cells of a rhesus macaque. Blood 107:3865-7, 2006.

Zong Y, Zhou S, Sorrentino BP. Reply to Rumpold et al. Leukemia 20:144-45, 2006.

Zong Y, Zhou S, Sorrentino BP. Loss of P-glycoprotein expression in hematopoietic stem cells does not improve responses to imantinib in a murine model of chronic myelogenous leukemia. Leukemia 19:1590-6, 2005.

Stepanova L, Sorrentino BP. A limited role for p16Ink4a and p19Arf in the loss of hematopoietic stem cells during proliferative stress. Blood 106:827-32, 2005.

McGaha TL, Sorrentino B, Ravetch JV. Restoration of tolerance in lupus by targeted inhibitory receptor expression. Science 307:590-3, 2005.

Zhou S, Yong Y, Ney PA, Nair G, Stewart CF, Sorrentino BP. Increased expression of the Abcg2 transporter during erythroid maturation plays a role in decreasing cellular protoporphyrin IX levels.Blood 105:2571-6. 2005.

Ozvegy-Laczka C, Varady G, Koblos G, Ujhelly O, Cervenak J, Schuetz JD, Sorrentino BP, Koomen GJ, Varadi A, Nemet K, Sarkadi B. Function-dependent Conformational Changes of the ABCG2 Multidrug Transporter Modify its Interaction with a Monoclonal Antibody on the Cell Surface. J Biol Chem 280: 4219-27, 2005.

Sorrentino BP. Clinical strategies for expansion of haematopoietic stem cells (review article). Nature Reviews Immunology 4:878-88, 2004.

Krishnamurthy P., Ross DD, Nakanishi T, Bailey-Dell K, Zhou S, Mercer KE, Sarkadi B, Sorrentino BP, Schuetz JD. The stem cell marker Bcrp/ABCG2 enhances hypoxic cell survival through interactions with heme. J Biol Chem 279: 24218-25. 2004.

Kiem HP, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Alder R, Kuramoto K, Calmels B, Bonifacino A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Neinhuis A, Blau CA, Andrews RG, Donahue RE, Dunbar CE. Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia. Molecular Therapy 9:389-95, 2004.

Persons DA, Allay JA, Bonifacino A, Lu T, Agricola B, Metzger ME, Donahue RE, Dunbar CE,Sorrentino BP. Transient In Vivo Selection of Transduced Peripheral Blood Cells Using Antifolate Drug Selection in Rhesus Macaques Transplanted with Hematopoietic Stem Cells Expressing Dihydrofolate Reductase Vectors. Blood 103:796-803, 2004.

Zhou S, Zong Y, Lu T, Sorrentino BP. Hematopoietic cells from mice that are deficient in both Bcrp1/Abcg2 and Mdr1a/1b develop normally but are sensitized to mitoxantrone. Biotechniques 35:1248-52, 2003.

Sawai N, Persons DA, Zhou S, Lu T, Sorrentino BP. Reduction in Hematopoietic Stem Cell Numbers with In Vivo Drug Selection Can Be Partially Abrogated by HOXB4 Gene Expression. Molecular Therapy 8:376-84, 2003.

Persons DA, Allay ER, Sawai N, Brent TP, Nienhuis AW, Sorrentino BP. Successful Treatment of Murine ß-Thalassemia by In Vivo Selection of Genetically-Modified, Drug-Resistant Hematopoietic Stem Cells. Blood 102:506-513, 2003.

Zhou S, Morris JJ, Barnes Y, Lan L, Schuetz JD, Sorrentino BP. 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 USA 99:12339-12344, 2002.

Abbott BL, Colapietro A-M, Barnes Y, Marini F, Andreeff M, Sorrentino BP. Low levels of ABCG2 expression in adult AML blast samples. Blood 100:4594-601, 2002.

Sorrentino, BP. Gene therapy to protect hamatopoietic cells from cytotoxic cancer drugs (review article). Nature Reviews, Cancer 2:431-441, 2002. 

Bunting KD, Bradley HL, Hawley TS, Moriggl R, Sorrentino BP, Ihle JN. Reduced lymphomyeloid repopulating activity from adult bone marrow and fetal liver of mice lacking expression of STAT 5. Blood 99:479-87, 2002.

Zhou S, Schuetz JD, Bunting KD, Colapietro A-M, Sampath J, Morris JJ, Lagutina I, Grosveld GC, Osawa M, Nakauchi H, Sorrentino BP. 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 7:1028-1034, 2001.

Donahue RE, Sorrentino BP, Hawley RG, An DS, Chen IS, Wersto RP. Fibronectin fragment CH-296 inhibits apoptosis and enhances ex vivo gene transfer by murine retrovirus and human lentivirus vectors independent of viral tropism in nonhuman primate CD34(+) cells. Molecular Therapy 3:359-67, 2001.

Sellers SE, Tisdale JF, Agricola BA, Metzger ME, Donahue RE, Dunbar CE Sorrentino BP. The effect of multidrug-resistance 1 gene versus neo transduction on ex vivo and in vivo expansion of rhesus macaque hematopoietic repopulating cells. Blood 97:1888-91, 2001.

Sawai N, Zhou S, Vanin EF, Houghton P, Brent TP, Sorrentino BP. Protection and in vivo selection of hematopoietic stem cells using temozolomide, O6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Molecular Therapy 3:78-87, 2001.

Bunting KD, Lu T, Kelly PF, Sorrentino BP. Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation.Human Gene Therapy 11:2353-2364, 2000.

Giogianni F, Bridson PK, Sorrentino BP, Pohl J, Blakley RL. Inactivation of aldophosphamide by human aldehyde dehyrdrogenase isoenzyme 3. Biochemical Pharmacology 60:325-338, 2000.

Bunting KD, Zhou S, Lu T, Sorrentino BP. Enforced P-glycoprotein pump function in murine bone marrow cells results in expansion of SP cells in vitro and repopulating cells in vivo. Blood 96:902-909, 2000.

Patel DH, Allay JA, Belt JA, Sorrentino BP. Retroviral transfer of the hENT2 nucleoside transporter cDNA confers broad-spectrum antifolate resistance in murine bone marrow cells. Blood 95:2356-2363, 2000.

Donahue RE, Wersto RP, Allay JA, Agricola BA, Metzger ME,, Nienhuis AW, Persons DA, Sorrentino BP. High levels of lymphoid expression of enhanced green fluorescent protein in non-human primates transplanted with cytokine mobilized peripheral blood CD34+ cells. Blood 95:445-452, 2000.

Last update: June 2016