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

Experimental Hematology

Volumn 36, Issue 8, 2008, Pages 957-964

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

^a GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

^b Medical Mannheim Center   (Germany)

^c UNIVERSITY OF HEIDELBERG   (Germany)

^d Paracelsus Klinik am Schillergarten   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; GREEN FLUORESCENT PROTEIN; IMATINIB; PARVOVIRUS VECTOR; PEPTIDE LIBRARY;

ACUTE GRANULOCYTIC LEUKEMIA; ARTICLE; CANCER GENE THERAPY; CHRONIC MYELOID LEUKEMIA; CONTROLLED STUDY; EMBRYO; FEMALE; GENE TRANSFER; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; LEUKEMIA CELL LINE; PERIPHERAL BLOOD STEM CELL; PRIORITY JOURNAL; TARGET CELL; VIRUS CAPSID; VIRUS MUTANT; VIRUS RECOMBINANT;

CAPSID; CELL LINE; DEPENDOVIRUS; DIRECTED MOLECULAR EVOLUTION; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELLS; HUMANS; LEUKEMIA; MUTAGENESIS; PEPTIDE LIBRARY; RECOMBINANT PROTEINS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION;

ADENO-ASSOCIATED VIRUS; MIRIDAE;

EID: 47349124310     PISSN: 0301472X     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.exphem.2008.03.007     Document Type: Article

References (58)

1. Aiuti A., Slavin S., Aker M., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (2002) 2410-2413
2. Bank A., Dorazio R., and Leboulch P. A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia. Ann N Y Acad Sci 1054 (2005) 308-316
3. Cavazzana-Calvo M., Hacein-Bey S., de Saint B.G., et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288 (2000) 669-672
4. Hanawa H., Hargrove P.W., Kepes S., et al. Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia. Blood 104 (2004) 2281-2290
5. Haneline L.S., Li X., Ciccone S.L., 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 101 (2003) 1299-1307
6. Levasseur D.N., Ryan T.M., Pawlik K.M., and Townes T.M. Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 102 (2003) 4312-4319
7. Liu J.M., Kim S., Read E.J., et al. Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC). Hum Gene Ther 10 (1999) 2337-2346
8. Malech H.L., Maples P.B., Whiting-Theobald N., et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci U S A 94 (1997) 12133-12138
9. May C., Rivella S., Callegari J., et al. Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin. Nature 406 (2000) 82-86
10. Pawliuk R., Westerman K.A., Fabry M.E., et al. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294 (2001) 2368-2371
11. Puthenveetil G., Scholes J., Carbonell D., et al. Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector. Blood 104 (2004) 3445-3453
12. Hacein-Bey-Abina S., Von K.C., Schmidt M., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
13. Kohn D.B., Sadelain M., Dunbar C., et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 8 (2003) 180-187
14. Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science 299 (2003) 320
15. McPhee S.W., Janson C.G., Li C., et al. Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 8 (2006) 577-588
16. Conrad C.K., Allen S.S., Afione S.A., et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther 3 (1996) 658-668
17. Blacklow N.R., Hoggan M.D., Sereno M.S., et al. A seroepidemiologic study of adenovirus-associated virus infection in infants and children. Am J Epidemiol 94 (1971) 359-366
18. Duan D., Sharma P., Dudus L., et al. Formation of adeno-associated virus circular genomes is differentially regulated by adenovirus E4 ORF6 and E2a gene expression. J Virol 73 (1999) 161-169
19. Flotte T.R. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. Curr Gene Ther 5 (2005) 361-366
20. McCarty D.M., Young Jr. S.M., and Samulski R.J. Integration of adeno-associated virus (AAV) and recombinant AAV vectors. Annu Rev Genet 38 (2004) 819-845
21. Miller D.G., Trobridge G.D., Petek L.M., et al. Large-scale analysis of adeno-associated virus vector integration sites in normal human cells. J Virol 79 (2005) 11434-11442
22. Berlinghoff S., Veldwijk M.R., Laufs S., et al. Susceptibility of mesothelioma cell lines to adeno-associated virus 2 vector-based suicide gene therapy. Lung Cancer 46 (2004) 179-186
23. Veldwijk M.R., Berlinghoff S., Laufs S., et al. Suicide gene therapy of sarcoma cell lines using recombinant adeno-associated virus 2 vectors. Cancer Gene Ther 11 (2004) 577-584
24. Aitken M.L., Moss R.B., Waltz D.A., et al. A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease. Hum Gene Ther 12 (2001) 1907-1916
25. Flotte T., Carter B., Conrad C., et al. A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 7 (1996) 1145-1159
26. Flotte T.R., Brantly M.L., Spencer L.T., et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum Gene Ther 15 (2004) 93-128
27. Kay M.A., Manno C.S., Ragni M.V., et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24 (2000) 257-261
28. Manno C.S., Chew A.J., Hutchison S., et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101 (2003) 2963-2972
29. Snyder R.O., and Francis J. Adeno-associated viral vectors for clinical gene transfer studies. Curr Gene Ther 5 (2005) 311-321
30. Wagner J.A., Nepomuceno I.B., Messner A.H., et al. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum Gene Ther 13 (2002) 1349-1359
31. Veldwijk M.R., Schiedlmeier B., Kleinschmidt J.A., Zeller W.J., and Fruehauf S. Superior gene transfer into solid tumour cells than into human mobilised peripheral blood progenitor cells using helpervirus-free adeno-associated viral vector stocks. Eur J Cancer 35 (1999) 1136-1142
32. Veldwijk M.R., Fruehauf S., Schiedlmeier B., Kleinschmidt J.A., and Zeller W.J. Differential expression of a recombinant adeno-associated virus 2 vector in human CD34(+) cells and breast cancer cells. Cancer Gene Ther 7 (2000) 597-604
33. Alexander I.E., Russell D.W., and Miller A.D. Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results. Hum Gene Ther 8 (1997) 1911-1920
34. Hargrove P.W., Vanin E.F., Kurtzman G.J., and Nienhuis A.W. High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3′ gamma globin gene regulatory element and integrates as tandem copies in erythroid cells. Blood 89 (1997) 2167-2175
35. Malik P., McQuiston S.A., Yu X.J., et al. Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. J Virol 71 (1997) 1776-1783
36. Nathwani A.C., Hanawa H., Vandergriff J., et al. Efficient gene transfer into human cord blood CD34+ cells and the CD34+. Gene Ther 7 (2000) 183-195
37. Zhou S.Z., Cooper S., Kang L.Y., et al. Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J Exp Med 179 (1994) 1867-1875
38. Ponnazhagan S., Mukherjee P., Wang X.S., 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 71 (1997) 8262-8267
39. Chatterjee S., Li W., Wong C.A., et al. Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors. Blood 93 (1999) 1882-1894
40. Tan M., Qing K., Zhou S., Yoder M.C., and Srivastava A. Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted long-term expression of the human beta-globin gene in hematopoietic cells from homozygous beta-thalassemic mice. Mol Ther 3 (2001) 940-946
41. Santat L., Paz H., Wong C., et al. Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice. Proc Natl Acad Sci U S A 102 (2005) 11053-11058
42. Muller O.J., Kaul F., Weitzman M.D., et al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 21 (2003) 1040-1046
43. Summerford C., and Samulski R.J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72 (1998) 1438-1445
44. Kern A., Schmidt K., Leder C., et al. Identification of a heparin-binding motif on adeno-associated virus type 2 capsids. J Virol 77 (2003) 11072-11081
45. Work L.M., Buning H., Hunt E., et al. Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses. Mol Ther 13 (2006) 683-693
46. White S.J., Nicklin S.A., Buning H., et al. Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors. Circulation 109 (2004) 513-519
47. Waterkamp D.A., Muller O.J., Ying Y., Trepel M., and Kleinschmidt J.A. Isolation of targeted AAV2 vectors from novel virus display libraries. J Gene Med 8 (2006) 1307-1319
48. Work L.M., Nicklin S.A., Brain N.J., et al. Development of efficient viral vectors selective for vascular smooth muscle cells. Mol Ther 9 (2004) 198-208
49. Salvetti A., Oreve S., Chadeuf G., et al. Factors influencing recombinant adeno-associated virus production. Hum Gene Ther 9 (1998) 695-706
50. Mahon F.X., Deininger M.W., Schultheis B., et al. Selection and characterization of BCR-ABL positive cell lines with differential sensitivity to the tyrosine kinase inhibitor STI571: diverse mechanisms of resistance. Blood 96 (2000) 1070-1079
51. Zolotukhin S., Byrne B.J., Mason E., et al. Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther 6 (1999) 973-985
52. Veldwijk M.R., Topaly J., Laufs S., et al. Development and optimization of a real-time quantitative PCR-based method for the titration of AAV-2 vector stocks. Mol Ther 6 (2002) 272-278
53. Schiedlmeier B., Wermann K., Kuhlcke K., et al. Human multidrug resistance-1 gene transfer to long-term repopulating human mobilized peripheral blood progenitor cells. Bone Marrow Transplant 25 Suppl 2 (2000) S118-S124
54. Schiedlmeier B., Kuhlcke K., Eckert H.G., et al. Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood 7progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice. Blood 95 (2000) 1237-1248
55. Fruehauf S., Veldwijk M.R., Berlinghoff S., et al. Gene therapy for sarcoma. Cells Tissues Organs 172 (2002) 133-144
56. Scherr M., Battmer K., Blomer U., et al. Lentiviral gene transfer into peripheral blood-derived CD34+ NOD/SCID-repopulating cells. Blood 99 (2002) 709-712
57. Geronimi F., Richard E., Redonnet-Vernhet I., et al. Highly efficient lentiviral gene transfer in CD34+ and CD34+/38-/lin- cells from mobilized peripheral blood after cytokine prestimulation. Stem Cells 21 (2003) 472-480
58. Alexander I.E., Russell D.W., and Miller A.D. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol 68 (1994) 8282-8287