Long-term in vivo monitoring of mouse and human hematopoietic stem cell engraftment with a human positron emission tomography reporter gene

Proceedings of the National Academy of Sciences of the United States of America

Volumn 110, Issue 5, 2013, Pages 1857-1862

  McCracken, Melissa N ^a   Gschweng, Eric H ^b   Nair Gill, Evan ^a   McLaughlin, Jami ^b   Cooper, Aaron R ^b   Riedinger, Mireille ^a   Cheng, Donghui ^b   Nosala, Christopher ^b   Kohn, Donald B ^b   Witte, Owen N ^a,b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Gene therapy; Molecular imaging

Indexed keywords

CLEVUDINE; CLEVUDINE F 18; THYMIDINE DERIVATIVE; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; BLOOD CELL COUNT; CELL DIFFERENTIATION; CELL LINEAGE; CELL MIGRATION; CONTROLLED STUDY; GENE EXPRESSION; GENE MUTATION; HDCK3MUT GENE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IN VIVO STUDY; MOUSE; NONHUMAN; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; REPORTER GENE;

ANIMALS; ARABINOFURANOSYLURACIL; BLOTTING, WESTERN; CELL LINE, TUMOR; DEOXYCYTIDINE KINASE; FEMALE; FLUORINE RADIOISOTOPES; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; IMMUNOHISTOCHEMISTRY; INTERLEUKIN RECEPTOR COMMON GAMMA SUBUNIT; KAPLAN-MEIER ESTIMATE; LUMINESCENT PROTEINS; MICE; MICE, INBRED C57BL; MICE, INBRED NOD; MICE, INBRED STRAINS; MICE, KNOCKOUT; MICE, SCID; MUTATION; POSITRON-EMISSION TOMOGRAPHY; THYMUS GLAND; TIME FACTORS; TRANSPLANTATION, HETEROLOGOUS;

EID: 84873191375     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1221840110     Document Type: Article

References (45)

1. Sng J, Lufkin T (2012) Emerging stem cell therapies: Treatment, safety, and biology. Stem Cells Int 2012:521343.
2. Herschman HR (2004) PET reporter genes for noninvasive imaging of gene therapy, cell tracking and transgenic analysis. Crit Rev Oncol Hematol 51(3):191-204. (Pubitemid 39158688)
3. Serganova I, Ponomarev V, Blasberg R (2007) Human reporter genes: Potential use in clinical studies. Nucl Med Biol 34(7):791-807. (Pubitemid 47503822)
4. Gyurkocza B, Rezvani A, Storb RF (2010) Allogeneic hematopoietic cell transplantation: The state of the art. Expert Rev Hematol 3(3):285-299.
5. Kohn DB (2010) Update on gene therapy for immunodeficiencies. Clin Immunol 135(2):247-254.
6. Gambhir SS, et al. (2000) A mutant herpes simplex virus type 1 thymidine kinase reporter gene shows improved sensitivity for imaging reporter gene expression with positron emission tomography. Proc Natl Acad Sci USA 97(6):2785-2790. (Pubitemid 30159249)
7. Tjuvajev JG, et al. (1998) Imaging herpes virus thymidine kinase gene transfer and expression by positron emission tomography. Cancer Res 58(19):4333-4341. (Pubitemid 28450034)
8. Berger C, Flowers ME, Warren EH, Riddell SR (2006) Analysis of transgene-specific immune responses that limit the in vivo persistence of adoptively transferred HSV-TK-modified donor T cells after allogeneic hematopoietic cell transplantation. Blood 107(6):2294-2302. (Pubitemid 43345547)
9. Riddell SR, et al. (1996) T-cell mediated rejection of gene-modified HIV-specific cytotoxic T lymphocytes in HIV-infected patients. Nat Med 2(2):216-223. (Pubitemid 26057556)
10. Traversari C, et al. (2007) The potential immunogenicity of the TK suicide gene does not prevent full clinical benefit associated with the use of TK-transduced donor lymphocytes in HSCT for hematologic malignancies. Blood 109(11):4708-4715. (Pubitemid 46827762)
11. Mercier-Letondal P, et al. (2008) Early immune response against retrovirally transduced herpes simplex virus thymidine kinase-expressing gene-modified T cells coinfused with a T cell-depleted marrow graft: An altered immune response? Hum Gene Ther 19(9):937-950.
12. Oliveira G, Greco R, Lupo-Stanghellini MT, Vago L, Bonini C (2012) Use of TK-cells in haploidentical hematopoietic stem cell transplantation. Curr Opin Hematol 19(6):427-433.
13. Campbell DO, et al. (2012) Structure-guided engineering of human thymidine kinase 2 as a positron emission tomography reporter gene for enhanced phosphorylation of a non-natural thymidine analog reporter probe. J Biol Chem 287(1):446-454.
14. Ponomarev V, et al. (2007) A human-derived reporter gene for noninvasive imaging in humans: Mitochondrial thymidine kinase type 2. J Nucl Med 48(5):819-826. (Pubitemid 47604984)
15. Likar Y, et al. (2010) A new pyrimidine-specific reporter gene: A mutated human deoxycytidine kinase suitable for PET during treatment with acycloguanosine-based cytotoxic drugs. J Nucl Med 51(9):1395-1403.
16. Choi O, et al. (2012) A deficiency in nucleoside salvage impairs murine lymphocyte development, homeostasis, and survival. J Immunol 188(8):3920-3927.
17. Toy G, et al. (2010) Requirement for deoxycytidine kinase in T and B lymphocyte development. Proc Natl Acad Sci USA 107(12):5551-5556.
18. Sabini E, Ort S, Monnerjahn C, Konrad M, Lavie A (2003) Structure of human dCK suggests strategies to improve anticancer and antiviral therapy. Nat Struct Biol 10(7):513-519. (Pubitemid 36792901)
19. Hazra S, Sabini E, Ort S, Konrad M, Lavie A (2009) Extending thymidine kinase activity to the catalytic repertoire of human deoxycytidine kinase. Biochemistry 48(6):1256-1263.
20. Iyidogan P, Lutz S (2008) Systematic exploration of active site mutations on human deoxycytidine kinase substrate specificity. Biochemistry 47(16):4711-4720. (Pubitemid 351555492)
21. Radu CG, et al. (2008) Molecular imaging of lymphoid organs and immune activation by positron emission tomography with a new [18F]-labeled 2'-deoxycytidine analog. Nat Med 14(7):783-788. (Pubitemid 351951515)
22. Austin WR, et al. (2012) Nucleoside salvage pathway kinases regulate hematopoiesis by linking nucleotide metabolism with replication stress. J Exp Med 209(12):2215-2228.
23. Jordheim LP, et al. (2004) Characterization of a gemcitabine-resistant murine leukemic cell line: Reversion of in vitro resistance by a mononucleotide prodrug. Clin Cancer Res 10(16):5614-5621. (Pubitemid 39100503)
24. Baum C, et al. (2003) Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 101(6):2099-2114. (Pubitemid 36302043)
25. Drize N, Chertkov J, Sadovnikova E, Tiessen S, Zander A (1997) Long-term maintenance of hematopoiesis in irradiated mice by retrovirally transduced peripheral blood stem cells. Blood 89(5):1811-1817. (Pubitemid 27097478)
26. Stewart FM, Crittenden RB, Lowry PA, Pearson-White S, Quesenberry PJ (1993) Longterm engraftment of normal and post-5-fluorouracil murine marrow into normal nonmyeloablated mice. Blood 81(10):2566-2571. (Pubitemid 23147558)
27. Kang E, et al. (2001) In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting. Hum Gene Ther 12(13):1663-1672. (Pubitemid 32911736)
28. Zavidij O, et al. (2012) Stable long-term blood formation by stem cells in murine steady-state hematopoiesis. Stem Cells 30(9):1961-1970.
29. Gambhir SS, et al. (2000) Imaging transgene expression with radionuclide imaging technologies. Neoplasia 2(1-2):118-138. (Pubitemid 30123184)
30. Hallek M, Wanders L, Strohmeyer S, Emmerich B (1992) Thymidine kinase: a Tumor marker with prognostic value for non-Hodgkin's lymphoma and a broad range of potential clinical applications. Ann Hematol 65(1):1-5.
31. Baldwin SA, et al. (2004) The equilibrative nucleoside transporter family, SLC29. Pflugers Arch 447(5):735-743. (Pubitemid 38241459)
32. Gray JH, Owen RP, Giacomini KM (2004) The concentrative nucleoside transporter family, SLC28. Pflugers Arch 447(5):728-734. (Pubitemid 38241458)
33. Park CY, Majeti R, Weissman IL (2008) In vivo evaluation of human hematopoiesis through xenotransplantation of purified hematopoietic stem cells from umbilical cord blood. Nat Protoc 3(12):1932-1940.
34. Corrigan-Curay J, et al. (2012) 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.
35. Biffi A, et al. (2011) Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. Blood 117(20):5332-5339.
36. Gonzalez-Murillo A, Lozano ML, Montini E, Bueren JA, Guenechea G (2008) Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors. Blood 112(8):3138-3147.
37. Ronen K, et al. (2011) Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia. Mol Ther 19(7):1273-1286.
38. Bonini C, et al. (1997) HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 276(5319):1719-1724. (Pubitemid 27421983)
39. Ciceri F, et al. (2007) Antitumor effects of HSV-TK-engineered donor lymphocytes after allogeneic stem-cell transplantation. Blood 109(11):4698-4707. (Pubitemid 46827761)
40. Lupo-Stanghellini MT, et al. (2010) Clinical impact of suicide gene therapy in allogeneic hematopoietic stem cell transplantation. Hum Gene Ther 21(3):241-250.
41. Tiberghien P, et al. (1994) Ganciclovir treatment of herpes simplex thymidine kinasetransduced primary T lymphocytes: An approach for specific in vivo donor T-cell depletion after bone marrow transplantation? Blood 84(4):1333-1341.
42. Hébrard C, Dumontet C, Jordheim LP (2009) Development of gene therapy in association with clinically used cytotoxic deoxynucleoside analogues. Cancer Gene Ther 16(7):541-550.
43. Burris HA, 3rd, et al. (1997) Improvements in survival and clinical benefit with gemcitabine as first-line therapy for patients with advanced pancreas cancer: A randomized trial. J Clin Oncol 15(6):2403-2413. (Pubitemid 27251144)
44. Hakenberg J, et al. (2003) MAPPP: MHC class I antigenic peptide processing prediction. Appl Bioinformatics 2(3):155-158.
45. Reche PA, Glutting JP, Reinherz EL (2002) Prediction of MHC class I binding peptides using profile motifs. Hum Immunol 63(9):701-709. (Pubitemid 34983301)