Evaluation of engraftment and immunological tolerance after reduced intensity conditioning in a rhesus hematopoietic stem cell gene therapy model

Gene Therapy - Tập 21 Số 2 - Trang 148-157 - 2014
Naoya Uchida1, R. Patrick Weitzel2, Molly Evans2, R Green2, Aylin Bonifacino3, Allen E. Krouse4, Mark E. Metzger4, Matthew M. Hsieh2, Robert E. Donahue4, John F. Tisdale2
1Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
2Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, USA
3Hematology Branch, NHLBI, NIH, 5 Research Court, Rockville, MD, USA.
4Hematology Branch, NHLBI, NIH, 5 Research Court, Rockville, USA

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Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447–458.

Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410–2413.

Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010; 467: 318–322.

Hacein-Bey-Abina S, von Kalle C, Schmidt M, Le Deist F, Wulffraat N, McIntyre E et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348: 255–256.

Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, Glimm H et al. Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J Clin Invest 2007; 117: 2241–2249.

Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006; 12: 401–409.

Copelan EA . Hematopoietic stem-cell transplantation. N Engl J Med 2006; 354: 1813–1826.

Hsieh MM, Kang EM, Fitzhugh CD, Link MB, Bolan CD, Kurlander R et al. Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med 2009; 361: 2309–2317.

Storb RF, Champlin R, Riddell SR, Murata M, Bryant S, Warren EH . Non-myeloablative transplants for malignant disease. Hematology Am Soc Hematol Educ Program 2001: 375–391.

Antin JH . Reduced-intensity stem cell transplantation: ‘...whereof a little More than a little is by much too much.’ King Henry IV, part 1, I, 2. Hematology Am Soc Hematol Educ Program 2007: 47–54.

Uchida N, Bonifacino A, Krouse AE, Metzger ME, Csako G, Lee-Stroka A et al. Accelerated lymphocyte reconstitution and long-term recovery after transplantation of lentiviral-transduced rhesus CD34(+) cells mobilized by G-CSF and plerixafor. Exp Hematol 2011; 39: 795–805.

Uchida N, Hargrove PW, Lap CJ, Evans ME, Phang O, Bonifacino AC et al. High-efficiency transduction of rhesus hematopoietic repopulating cells by a modified HIV1-based lentiviral vector. Mol Ther 2012; 20: 1882–1892.

Uchida N, Washington KN, Hayakawa J, Hsieh MM, Bonifacino AC, Krouse AE et al. Development of a human immunodeficiency virus type 1-based lentiviral vector that allows efficient transduction of both human and rhesus blood cells. J Virol 2009; 83: 9854–9862.

Heim DA, Hanazono Y, Giri N, Wu T, Childs R, Sellers SE et al. Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model. Mol Ther 2000; 1: 533–544.

Kung SK, An DS, Bonifacino A, Metzger ME, Ringpis GE, Mao SH et al. Induction of transgene-specific immunological tolerance in myeloablated nonhuman primates using lentivirally transduced CD34+ progenitor cells. Mol Ther 2003; 8: 981–991.

Nienhuis AW . Development of gene therapy for blood disorders. Blood 2008; 111: 4431–4444.

Persons DA . Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders. Hematology Am Soc Hematol Educ Program 2009; 1: 690–697.

Bunn HF . Pathogenesis and treatment of sickle cell disease. N Engl J Med 1997; 337: 762–769.

Otieno S, Bloor A, Karol R, Reichlin M, Noble RW . Specific antibodies to hemoglobin A1 (anti-Glu) and hemoglobin S (anti-Val) in the guinea pig: immunologic and structural correlations. J Immunol 1978; 121: 2458–2462.

Uchida N, Hsieh MM, Hayakawa J, Madison C, Washington KN, Tisdale JF . Optimal conditions for lentiviral transduction of engrafting human CD34(+) cells. Gene Therapy 2011; 18: 1078–1086.

Uchida N, Washington KN, Lap CJ, Hsieh MM, Tisdale JF . Chicken HS4 insulators have minimal barrier function among progeny of human hematopoietic cells transduced with an HIV1-based lentiviral vector. Mol Ther 2010; 19: 133–139.

Hanawa H, Persons DA, Nienhuis AW . Mobilization and mechanism of transcription of integrated self-inactivating lentiviral vectors. J Virol 2005; 79: 8410–8421.

Uchida N, Hanawa H, Yamamoto M, Shimada T . The chicken HS4 core insulator blocks promoter interference in lentiviral vectors. Hum Gene Ther Methods 2013; 24: 117–124.

Hanawa H, Hematti P, Keyvanfar K, Metzger ME, Krouse A, Donahue RE et al. Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system. Blood 2004; 103: 4062–4069.

Uchida N, Hanawa H, Dan K, Inokuchi K, Shimada T . Leukemogenesis of b2a2-type p210 BCR/ABL in a bone marrow transplantation mouse model using a lentiviral vector. J Nippon Med Sch 2009; 76: 134–147.

Tisdale JF, Hanazono Y, Sellers SE, Agricola BA, Metzger ME, Donahue RE et al. Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 1998; 92: 1131–1141.

Uchida N, Hsieh MM, Washington KN, Tisdale JF . Efficient transduction of human hematopoietic repopulating cells with a chimeric HIV1-based vector including SIV capsid. Exp Hematol 2013; 41: 779–788.

Hanazono Y, Brown KE, Handa A, Metzger ME, Heim D, Kurtzman GJ et al. In vivo marking of rhesus monkey lymphocytes by adeno-associated viral vectors: direct comparison with retroviral vectors. Blood 1999; 94: 2263–2270.

Parry RV, Rumbley CA, Vandenberghe LH, June CH, Riley JL . CD28 and inducible costimulatory protein Src homology 2 binding domains show distinct regulation of phosphatidylinositol 3-kinase, Bcl-xL, and IL-2 expression in primary human CD4 T lymphocytes. J Immunol 2003; 171: 166–174.

Pollok KE, Hanenberg H, Noblitt TW, Schroeder WL, Kato I, Emanuel D et al. High-efficiency gene transfer into normal and adenosine deaminase-deficient T lymphocytes is mediated by transduction on recombinant fibronectin fragments. J Virol 1998; 72: 4882–4892.

Zhou P, Lee J, Moore P, Brasky KM . High-efficiency gene transfer into rhesus macaque primary T lymphocytes by combining 32 degrees C centrifugation and CH-296-coated plates: effect of gene transfer protocol on T cell homing receptor expression. Hum Gene Ther 2001; 12: 1843–1855.

Deeks SG, Wagner B, Anton PA, Mitsuyasu RT, Scadden DT, Huang C et al. A phase II randomized study of HIV-specific T-cell gene therapy in subjects with undetectable plasma viremia on combination antiretroviral therapy. Mol Ther 2002; 5: 788–797.

Varela-Rohena A, Molloy PE, Dunn SM, Li Y, Suhoski MM, Carroll RG et al. Control of HIV-1 immune escape by CD8 T cells expressing enhanced T-cell receptor. Nat Med 2008; 14: 1390–1395.

Geraerts M, Willems S, Baekelandt V, Debyser Z, Gijsbers R . Comparison of lentiviral vector titration methods. BMC Biotechnol 2006; 6: 34.

Houter JVKD. Antibody response induced after intraocular viral gene addition therapy using adeno-associated virus, lentivirus, and adenovirus vectors with the GFP Transgene in dogs. ProQuest LLC Ann Arbor, MI, USA, 2009.