22
Views
1
CrossRef citations to date
0
Altmetric
SHORT COMMUNICATION

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

, , , , , , , & show all
Pages 107-112 | Received 10 Jun 2009, Accepted 17 Sep 2009, Published online: 24 Nov 2009

References

  • Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, . Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296:2410–3.
  • Cavazzana-Calvo M, Hacein-Bey S, de Saint BG, Gross F, Yvon E, Nusbaum P, . Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288:669–72.
  • Haneline LS, Li X, Ciccone SL, Hong P, Yang Y, Broxmeyer HE, . Retroviral-mediated expression of recombinant Fancc enhances the repopulating ability of Fancc-/- hematopoietic stem cells and decreases the risk of clonal evolution. Blood 2003; 101:1299–307.
  • 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; 102:4312–9.
  • Bozorgmehr F, Laufs S, Sellers SE, Roeder I, Zeller WJ, Dunbar CE, . 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; 25:2610–8.
  • Hacein-Bey-Abina S, Von KC, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, . LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302:415–9.
  • Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science 2003;299:320.
  • Mcphee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, . Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 2006; 8:577–88.
  • McCarty DM, Young SMJr, Samulski RJ. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 2004;38:819–45.
  • Sarkar R, Tetreault R, Gao G, Wang L, Bell P, Chandler R, . Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood 2004; 103:1253–60.
  • Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res 2005; 58:1143–7.
  • Veldwijk MR, Berlinghoff S, Laufs S, Hengge UR, Zeller WJ, Wenz F, . Suicide gene therapy of sarcoma cell lines using recombinant adeno-associated virus 2 vectors. Cancer Gene Ther 2004; 11:577–84.
  • Srivastava A. Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions. Hum Gene Ther 2005; 16:792–8.
  • Chatterjee S, Li W, Wong CA, Fisher-Adams G, Lu D, Guha M, . Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors. Blood 1999; 93:1882–94.
  • Ponnazhagan S, Mukherjee P, Wang XS, Qing K, Kube DM, Mah C, . 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; 71:8262–7.
  • Veldwijk MR, Fruehauf S, Schiedlmeier B, Kleinschmidt JA, Zeller WJ. Differential expression of a recombinant adeno-associated virus 2 vector in human CD34(+) cells and breast cancer cells. Cancer Gene Ther 2000; 7:597–604.
  • Muller OJ, Kaul F, Weitzman MD, Pasqualini R, Arap W, Kleinschmidt JA, . Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 2003; 21:1040–6.
  • Sellner L, Stiefelhagen M, Kleinschmidt JA, Laufs S, Wenz F, Fruehauf S, . 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; 36:957–64.
  • Stiefelhagen M, Sellner L, Kleinschmidt JA, Jauch A, Laufs S, Wenz F, . 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.
  • 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.
  • 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;1:137–47.
  • 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; 7:839–50.
  • Wang Z, Ma HI, Li J, Sun L, Zhang J, Xiao X. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther 2003; 10:2105–11.
  • 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; 7:183–95.
  • Santat L, Paz H, Wong C, Li L, Macer J, Forman S, . Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice. Proc Natl Acad Sci USA 2005;102: 11053–8.
  • 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-CSF-supported chemotherapy. Stem Cells 1998; 16:271–9.
  • Zhong L, Li W, Li Y, Zhao W, Wu J, Li B, . Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5. Hum Gene Ther 2006; 17:321–33.
  • Dupuy FP, Mouly E, Mesel-Lemoine M, Morel C, Abriol J, Cherai M, . 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; 7:1158–71.
  • 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; 96:3392–8.
  • Sirven A, Ravet E, Charneau P, Zennou V, Coulombel L, Guetard D, . 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;3:438–48.
  • Woods NB, Ooka A, Karlsson S. Development of gene therapy for hematopoietic stem cells using lentiviral vectors. Leukemia 2002;16:563–9.
  • Berlinghoff S, Veldwijk MR, Laufs S, Maser HP, Jauch A, Wenz F, . Susceptibility of mesothelioma cell lines to adeno-associated virus 2 vector-based suicide gene therapy. Lung Cancer 2004;46:179–86.
  • 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;23:3558–65.
  • Hütter G, Nowak D, Mossner M, Ganepola S, Müssig A, Allers K, . Long-term control of HIV by CCR5 Delta32/Delta32 stem-cell transplantation. N Engl J Med 2009;360:692–8.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.