Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34+ peripheral blood progenitor cells

Cytotherapy

Volumn 12, Issue 1, 2010, Pages 107-112

  Veldwijk, Marlon R ^a,b   Sellner, Leopold ^b,c   Stiefelhagen, Marius ^b   Kleinschmidt, Jürgen A ^b   Laufs, Stephanie ^a,b   Topaly, Julian ^c,d   Fruehauf, Stefan ^d   Zeller, W Jens ^b   Wenz, Frederik ^a  

^a UNIVERSITY OF HEIDELBERG   (Germany)

^b GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^c UNIVERSITY OF HEIDELBERG   (Germany)

^d Paracelsus Klinik   (Germany)

Author keywords

Adeno associated virus; Gene transfer; Peripheral blood progenitor cells; Pseudotyped virus

Indexed keywords

CD34 ANTIGEN; DOUBLE STRANDED DNA; GRANULOCYTE COLONY STIMULATING FACTOR; PARVOVIRUS VECTOR;

ADULT; ARTICLE; CLINICAL ARTICLE; DNA SYNTHESIS; FEMALE; GENE EXPRESSION; GENE TRANSFER; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; MALE; PERIPHERAL BLOOD STEM CELL; PRIORITY JOURNAL; PSEUDOTYPING; SEROTYPE; TRANSGENE; VIRUS CAPSID; VIRUS GENOME;

EID: 75449109000     PISSN: 14653249     EISSN: 14772566     Source Type: Journal    
DOI: 10.3109/14653240903348293     Document Type: Article

References (34)

1. 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
2. Cavazzana-Calvo M, Hacein-Bey S, de Saint BG, Gross F, Yvon E, Nusbaum P, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-672 (Pubitemid 30241569)
3. Haneline LS, Li X, Ciccone SL, Hong P, Yang Y, Broxmeyer HE, et al. Retroviral-mediated expression of recombinant Fancc enhances the repopulating ability of Fancc-/- hematopoietic stem cells and decreases the risk of clonal evolution. Blood 2003;101:1299-1307
4. Levasseur DN, Ryan TM, Pawlik KM, Townes TM. Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 2003;102:4312-4319
5. Bozorgmehr F, Laufs S, Sellers SE, Roeder I, Zeller WJ, Dunbar CE, et al. No evidence of clonal dominance in primates up to 4 years following transplantation of multidrug resistance 1 retrovirally transduced long-term repopulating cells. Stem Cells 2007;25:2610-2618
6. Hacein-Bey-Abina S, Von KC, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-419
7. Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science 2003;299:320.
8. McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, et al. Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 2006;8:577-588
9. McCarty DM, Young SM Jr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 2004;38:819-845
10. Sarkar R, Tetreault R, Gao G, Wang L, Bell P, Chandler R, et al. Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood 2004;103:1253-1260
11. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res 2005;58:1143-1147
12. Veldwijk MR, Berlinghoff S, Laufs S, Hengge UR, Zeller WJ, Wenz F, et al. Suicide gene therapy of sarcoma cell lines using recombinant adeno-associated virus 2 vectors. Cancer Gene Ther 2004;11:577-584 (Pubitemid 39079496)
13. Srivastava A. Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions. Hum Gene Ther 2005;16:792-798
14. Chatterjee S, Li W, Wong CA, Fisher-Adams G, Lu D, Guha M, et al. Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors. Blood 1999;93:1882-1894
15. Ponnazhagan S, Mukherjee P, Wang XS, Qing K, Kube DM, Mah C, et al. Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34- hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation. J Virol 1997;71:8262-8267
16. Veldwijk MR, Fruehauf S, Schiedlmeier B, Kleinschmidt JA, Zeller WJ. Differential expression of a recombinant adenoassociated virus 2 vector in human CD34(-) cells and breast cancer cells. Cancer Gene Ther 2000;7:597-604.
17. Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W, Kleinschmidt JA, et al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 2003;21:1040-1046
18. Sellner L, Stiefelhagen M, Kleinschmidt JA, Laufs S, Wenz F, Fruehauf S, et al. Generation of efficient human blood progenitor-targeted recombinant adeno-associated viral vectors (AAV) by applying an AAV random peptide library on primary human hematopoietic progenitor cells. Exp Hematol 2008;36:957-964
19. Stiefelhagen M, Sellner L, Kleinschmidt JA, Jauch A, Laufs S, Wenz F, et al. Application of a haematopoetic progenitor cell-targeted adeno-associated viral (AAV) vector established by selection of an AAV random peptide library on a leukaemia cell line. Genet Vaccines Ther 2008;6:12.
20. Grimm D, Kay MA. From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 2003;3:281-304.
21. Sanlioglu S, Monick MM, Luleci G, Hunninghake GW, Engelhardt JF. Rate limiting steps of AAV transduction and implications for human gene therapy. Curr Gene Ther 2001;1:137-147
22. Grimm D, Kay MA, Kleinschmidt JA. Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol Ther 2003;7:839-850
23. Wang Z, Ma HI, Li J, Sun L, Zhang J, Xiao X. Rapid and highly efficient transduction by double-stranded adenoassociated virus vectors in vitro and in vivo. Gene Ther 2003;10:2105-2111
24. Nathwani AC, Hanawa H, Vandergriff J, Kelly P, Vanin EF, Nienhuis AW. Efficient gene transfer into human cord blood CD34- cells and the CD34-. Gene Ther 2000;7:183-195
25. Santat L, Paz H, Wong C, Li L, Macer J, Forman S, et al. Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice. Proc Natl Acad Sci USA 2005;102: 11053-11058
26. Fruehauf S, Veldwijk MR, Kramer A, Haas R, Zeller WJ. Delineation of cell cycle state and correlation to adhesion molecule expression of human CD34- cells from steady-state bone marrow and peripheral blood mobilized following G-CSFsupported chemotherapy. Stem Cells 1998;16:271-279
27. Zhong L, Li W, Li Y, Zhao W, Wu J, Li B, et al. Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adenoassociated virus vector serotypes 1 through 5. Hum Gene Ther 2006;17:321-333
28. Dupuy FP, Mouly E, Mesel-Lemoine M, Morel C, Abriol J, Cherai M, et al. Lentiviral transduction of human hematopoietic cells by HIV-1- and SIV-based vectors containing a bicistronic cassette driven by various internal promoters. J Gene Med 2005;7:1158-1171
29. Salmon P, Kindler V, Ducrey O, Chapuis B, Zubler RH, Trono D. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 2000;96:3392-3398
30. Sirven A, Ravet E, Charneau P, Zennou V, Coulombel L, Guetard D, et al. Enhanced transgene expression in cord blood CD34(-)-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors. Mol Ther 2001;3:438-448
31. Woods NB, Ooka A, Karlsson S. Development of gene therapy for hematopoietic stem cells using lentiviral vectors. Leukemia 2002;16:563-569
32. Berlinghoff S, Veldwijk MR, Laufs S, Maser HP, Jauch A, Wenz F, et al. Susceptibility of mesothelioma cell lines to adeno-associated virus 2 vector-based suicide gene therapy. Lung Cancer 2004;46:179-186 (Pubitemid 39330412)
33. Porteus MH, Cathomen T, Weitzman MD, Baltimore D. Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks. Mol Cell Biol 2003;23: 3558-3565
34. Hutter G, Nowak D, Mossner M, Ganepola S, Mussig A, Allers K, et al. Long-term control of HIV by CCR5 Delta32/ Delta32 stem-cell transplantation. N Engl J Med 2009; 360:692-698