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Upregulated heme biosynthesis, an exploitable vulnerability in MYCN-driven leukemogenesis.

Fukuda Y, Wang Y, Lian S, Lynch J, Nagai S, Fanshawe B, Kandilci A, Janke LJ, Neale G, Fan Y, Sorrentino BP, Roussel MF, Grosveld G, Schuetz JD.

JCI Insight. 2017 Aug 3;2(15). pii: 92409. doi: 10.1172/jci.insight.92409. [Epub ahead of print]


The genomic landscape of pediatric and young adult T-lineage acute lymphoblastic leukemia.

Liu Y, Easton J, Shao Y, Maciaszek J, Wang Z, Wilkinson MR, McCastlain K, Edmonson M, Pounds SB, Shi L, Zhou X, Ma X, Sioson E, Li Y, Rusch M, Gupta P, Pei D, Cheng C, Smith MA, Auvil JG, Gerhard DS, Relling MV, Winick NJ, Carroll AJ, Heerema NA, Raetz E, Devidas M, Willman CL, Harvey RC, Carroll WL, Dunsmore KP, Winter SS, Wood BL, Sorrentino BP, Downing JR, Loh ML, Hunger SP, Zhang J, Mullighan CG.

Nat Genet. 2017 Aug;49(8):1211-1218. doi: 10.1038/ng.3909. Epub 2017 Jul 3.


Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.

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, Kardava L.

Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856. Erratum in: Sci Transl Med. 2016 Jun 1;8(341):341er5.


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

Zhou S, Fatima S, Ma Z, Wang YD, Lu T, Janke LJ, Du Y, Sorrentino BP.

Mol Ther. 2016 Jun;24(6):1090-1099. doi: 10.1038/mt.2016.55. Epub 2016 Mar 9.


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

Doyle MJ, Maher TJ, Li Q, Garry MG, Sorrentino BP, Martin CM.

Stem Cells Dev. 2016 Feb 1;25(3):277-84. doi: 10.1089/scd.2015.0272. Epub 2016 Jan 7.


ABCG2 Transporter Expression Impacts Group 3 Medulloblastoma Response to Chemotherapy.

Morfouace M, Cheepala S, Jackson S, Fukuda Y, Patel YT, Fatima S, Kawauchi D, Shelat AA, Stewart CF, Sorrentino BP, Schuetz JD, Roussel MF.

Cancer Res. 2015 Sep 15;75(18):3879-89. doi: 10.1158/0008-5472.CAN-15-0030. Epub 2015 Jul 21.


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

Felsburg PJ, De Ravin SS, Malech HL, Sorrentino BP, Burtner C, Kiem HP.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):50-6. doi: 10.1089/humc.2015.004. Epub 2015 Feb 24. Review.


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

Zhou S, Bonner MA, Wang YD, Rapp S, De Ravin SS, Malech HL, Sorrentino BP.

Hum Gene Ther Methods. 2015 Feb;26(1):4-12. doi: 10.1089/hgtb.2014.122. Epub 2015 Feb 5.


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

Yu H, Neale G, Zhang H, Lee HM, Ma Z, Zhou S, Forget BG, Sorrentino BP.

Blood. 2014 Sep 11;124(11):1737-47. doi: 10.1182/blood-2013-10-534735. Epub 2014 Jul 31.


Charting a clear path: the ASGCT Standardized Pathways Conference.

Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR.

Mol Ther. 2014 Jul;22(7):1235-1238. doi: 10.1038/mt.2014.95. No abstract available.


ATP-binding cassette transporter Abcg2 lineage contributes to the cardiac vasculature after oxidative stress.

Maher TJ, Ren Y, Li Q, Braunlin E, Garry MG, Sorrentino BP, Martin CM.

Am J Physiol Heart Circ Physiol. 2014 Jun 15;306(12):H1610-8. doi: 10.1152/ajpheart.00638.2013. Epub 2014 Apr 11.


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

Treanor LM, Zhou S, Janke L, Churchman ML, Ma Z, Lu T, Chen SC, Mullighan CG, Sorrentino BP.

J Exp Med. 2014 Apr 7;211(4):701-13. doi: 10.1084/jem.20122727. Epub 2014 Mar 31.


False-positive HIV PCR test following ex vivo lentiviral gene transfer treatment of X-linked severe combined immunodeficiency vector.

De Ravin SS, Gray JT, Throm RE, Spindler J, Kearney M, Wu X, Coffin JM, Hughes SH, Malderelli F, Sorrentino BP, Malech HL.

Mol Ther. 2014 Feb;22(2):244-245. doi: 10.1038/mt.2013.296. No abstract available.


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

Koldej RM, Carney G, Wielgosz MM, Zhou S, Zhan J, Sorrentino BP, Nienhuis AW.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):77-85. doi: 10.1089/humc.2012.244. Epub 2013 Jun 20.


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

Zhou S, Ma Z, Lu T, Janke L, Gray JT, Sorrentino BP.

PLoS One. 2013 Apr 23;8(4):e62333. doi: 10.1371/journal.pone.0062333. Print 2013.


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

Yasuda K, Cline C, Vogel P, Onciu M, Fatima S, Sorrentino BP, Thirumaran RK, Ekins S, Urade Y, Fujimori K, Schuetz EG.

Drug Metab Dispos. 2013 Apr;41(4):923-31. doi: 10.1124/dmd.112.050344. Epub 2013 Jan 8.


Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.

Greene MR, Lockey T, Mehta PK, Kim YS, Eldridge PW, Gray JT, Sorrentino BP.

Hum Gene Ther Methods. 2012 Oct;23(5):297-308. doi: 10.1089/hgtb.2012.150. Epub 2012 Nov 7.


Chemoprotection in brain tumor patients: another success for stem cell gene therapy.

Sorrentino BP.

Mol Ther. 2012 Aug;20(8):1485-7. doi: 10.1038/mt.2012.139. No abstract available.


Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy.

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.

Mol Ther. 2012 Jun;20(6):1084-94. doi: 10.1038/mt.2012.93. No abstract available.


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

Fatima S, Zhou S, Sorrentino BP.

Stem Cells. 2012 Feb;30(2):210-21. doi: 10.1002/stem.1002.


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

Telbisz Á, Hegedüs C, Özvegy-Laczka C, Goda K, Várady G, Takáts Z, Szabó E, Sorrentino BP, Váradi A, Sarkadi B.

Eur J Pharm Sci. 2012 Jan 23;45(1-2):101-9. doi: 10.1016/j.ejps.2011.10.021. Epub 2011 Nov 17.


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

Doyle MJ, Zhou S, Tanaka KK, Pisconti A, Farina NH, Sorrentino BP, Olwin BB.

J Cell Biol. 2011 Oct 3;195(1):147-63. doi: 10.1083/jcb.201103159. Epub 2011 Sep 26.


Identification of a novel, tissue-specific ABCG2 promoter expressed in pediatric acute megakaryoblastic leukemia.

Campbell PK, Zong Y, Yang S, Zhou S, Rubnitz JE, Sorrentino BP.

Leuk Res. 2011 Oct;35(10):1321-9. doi: 10.1016/j.leukres.2011.05.004.


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

Treanor LM, Volanakis EJ, Zhou S, Lu T, Sherr CJ, Sorrentino BP.

Blood. 2011 May 19;117(20):5453-62. doi: 10.1182/blood-2010-09-309831. Epub 2011 Mar 22.


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

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.

Blood. 2010 Aug 12;116(6):900-8. doi: 10.1182/blood-2009-10-250209. Epub 2010 May 10.


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

Jiang J, Yu H, Shou Y, Neale G, Zhou S, Lu T, Sorrentino BP.

Blood. 2010 Aug 5;116(5):711-9. doi: 10.1182/blood-2009-07-235341. Epub 2010 Apr 14.


Scl and stem cell quiescence.

Sorrentino BP.

Blood. 2010 Jan 28;115(4):751-2. doi: 10.1182/blood-2009-11-251264. No abstract available.


In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene.

Larochelle A, Choi U, Shou Y, Naumann N, Loktionova NA, Clevenger JR, Krouse A, Metzger M, Donahue RE, Kang E, Stewart C, Persons D, Malech HL, Dunbar CE, Sorrentino BP.

J Clin Invest. 2009 Jul;119(7):1952-63. doi: 10.1172/JCI37506. Epub 2009 Jun 8.


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

Cicero SA, Johnson D, Reyntjens S, Frase S, Connell S, Chow LM, Baker SJ, Sorrentino BP, Dyer MA.

Proc Natl Acad Sci U S A. 2009 Apr 21;106(16):6685-90. doi: 10.1073/pnas.0901596106. Epub 2009 Apr 3.


Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection.

Throm RE, Ouma AA, Zhou S, Chandrasekaran A, Lockey T, Greene M, De Ravin SS, Moayeri M, Malech HL, Sorrentino BP, Gray JT.

Blood. 2009 May 21;113(21):5104-10. doi: 10.1182/blood-2008-11-191049. Epub 2009 Mar 13.


Interaction with the 5D3 monoclonal antibody is regulated by intramolecular rearrangements but not by covalent dimer formation of the human ABCG2 multidrug transporter.

Ozvegy-Laczka C, Laczkó R, Hegedus C, Litman T, Várady G, Goda K, Hegedus T, Dokholyan NV, Sorrentino BP, Váradi A, Sarkadi B.

J Biol Chem. 2008 Sep 19;283(38):26059-70. doi: 10.1074/jbc.M803230200. Epub 2008 Jul 21.


Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials.

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, Wagner LM, Kelly P, Reeves L, Williams DA.

Mol Ther. 2007 Apr;15(4):782-91. Epub 2007 Feb 13.


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

Zong Y, Zhou S, Fatima S, Sorrentino BP.

J Biol Chem. 2006 Oct 6;281(40):29625-32. Epub 2006 Aug 1.


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

Shou Y, Ma Z, Lu T, Sorrentino BP.

Proc Natl Acad Sci U S A. 2006 Aug 1;103(31):11730-5. Epub 2006 Jul 24.


Genotoxicity of retroviral integration in hematopoietic cells.

Nienhuis AW, Dunbar CE, Sorrentino BP.

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


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

Tadjali M, Zhou S, Rehg J, Sorrentino BP.

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


Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque.

Seggewiss R, Pittaluga S, Adler RL, Guenaga FJ, Ferguson C, Pilz IH, Ryu B, Sorrentino BP, Young WS 3rd, Donahue RE, von Kalle C, Nienhuis AW, Dunbar CE.

Blood. 2006 May 15;107(10):3865-7. Epub 2006 Jan 26.


Reply to Rumpold et al.

Zong Y, Zhou S, Sorrentino BP.

Leukemia. 2006 Jan;20(1):144-145. No abstract available.


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

Ozvegy-Laczka C, Várady G, Köblös G, Ujhelly O, Cervenak J, Schuetz JD, Sorrentino BP, Koomen GJ, Váradi A, Német K, Sarkadi B.

J Biol Chem. 2005 Feb 11;280(6):4219-27. Epub 2004 Nov 22.


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

Zhou S, Zong Y, Ney PA, Nair G, Stewart CF, Sorrentino BP.

Blood. 2005 Mar 15;105(6):2571-6. Epub 2004 Nov 16.


Clinical strategies for expansion of haematopoietic stem cells.

Sorrentino BP.

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


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

Krishnamurthy P, Ross DD, Nakanishi T, Bailey-Dell K, Zhou S, Mercer KE, Sarkadi B, Sorrentino BP, Schuetz JD.

J Biol Chem. 2004 Jun 4;279(23):24218-25. Epub 2004 Mar 24.


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.

Persons DA, Allay JA, Bonifacino A, Lu T, Agricola B, Metzger ME, Donahue RE, Dunbar CE, Sorrentino BP.

Blood. 2004 Feb 1;103(3):796-803. Epub 2003 Aug 14.


Development of gene therapy for hemoglobin disorders.

Nienhuis AW, Hanawa H, Sawai N, Sorrentino BP, Persons DA.

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


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

Persons DA, Allay ER, Sawai N, Hargrove PW, Brent TP, Hanawa H, Nienhuis AW, Sorrentino BP.

Blood. 2003 Jul 15;102(2):506-13. Epub 2003 Mar 27.

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