RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy

Journal of Gene Medicine

Volumn 12, Issue 9, 2010, Pages 766-778

  Hu, Chuhong ^a   Busuttil, Ronald W ^a   Lipshutz, Gerald S ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adeno associated virus; Gene therapy; Neonate; Serotype rh10

Indexed keywords

ADENOVIRUS VECTOR; LUCIFERASE;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS RH10; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; COPY NUMBER VARIATION; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; MITOSIS; MOUSE; NEWBORN; NONHUMAN; ORGAN GROWTH; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN LOCALIZATION; SEROTYPE; TRANSGENE;

ANIMALS; ANIMALS, NEWBORN; DEPENDOVIRUS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; LUCIFERASES; MICE; SEROTYPING; TISSUE DISTRIBUTION; TRANSGENES;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MUS;

EID: 77956628142     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1496     Document Type: Article

References (55)

1. Grimm D, Zhou S, Nakai H, et al. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 2003; 102: 2412-2419.
2. Song Y, Lou HH, Boyer JL, et al. Functional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicing. Hum Gene Ther 2009; 20: 267-281.
3. Katakura S, Jennings K, Watanabe S, et al. Recombinant adeno-associated virus preferentially transduces human, compared to mouse, synovium: implications for arthritis therapy. Mod Rheumatol 2004; 14: 18-24. (Pubitemid 40796155)
4. Simonelli F, Maguire AM, Testa F, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 2010; 18: 643-650.
5. Arruda VR, Stedman HH, Haurigot V, et al. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood 2010; 115: 4678-4688.
6. Foust KD, Wang X, McGovern VL, et al. Rescue of the spinal muscular atrophy phenotype in amouse model by early postnatal delivery of SMN. Nat Biotechnol 2010; 28: 271-274.
7. Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008; 16: 1073-1080.
8. Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther 2008; 16: 1710-1718.
9. Palomeque J, Chemaly ER, Colosi P, et al. Efficiency of eight different AAV serotypes in transducing rat myocardium in vivo. Gene Ther 2007; 14: 989-997.
10. Lipshutz GS, Gruber CA, Cao Y, Hardy J, Contag CH, Gaensler KM. In utero delivery of adeno-associated viral vectors: intraperitoneal gene transfer produces long-term expression. Mol Ther 2001; 3: 284-292. (Pubitemid 32514643)
11. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-2232.
12. Nguyen AT, Dow AC, Kupiec-Weglinski J, Busuttil RW, Lipshutz GS. Evaluation of gene promoters for liver expression by hydrodynamic gene transfer. J Surg Res 2008; 148: 60-66.
13. Wu JC, Sundaresan G, Iyer M, Gambhir SS. Noninvasive optical imaging of firefly luciferase reporter gene expression in skeletal muscles of living mice. Mol Ther 2001; 4: 297-306. (Pubitemid 33040513)
14. Cunningham SC, Dane AP, Spinoulas A, Logan GJ, Alexander IE. Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther 2008; 16: 1081-1088.
15. Sabatino DE, Mackenzie TC, Peranteau W, et al. Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Mol Ther 2007; 15: 1677-1685.
16. Kyosen SO, Iizuka S, Kobayashi H, et al. Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction. Gene Ther 2010; 17: 521-530.
17. Pacak CA, Mah CS, Thattaliyath BD, et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res 2006; 99: E3-E9.
18. Waddington SN, Kennea NL, Buckley SM, Gregory LG, Themis M, Coutelle C. Fetal and neonatal gene therapy: benefits and pitfalls. Gene Ther 2004; 11(Suppl 1): S92-S97. (Pubitemid 39359453)
19. Ponder KP. Immunology of neonatal gene transfer. Curr Gene Ther 2007; 7: 403-410.
20. Takahashi M, Ilan Y, Chowdhury NR, Guida J, Horwitz M, Chowdhury JR. Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period. J Biol Chem 1996; 271: 26536-26542.
21. Xu L, Haskins ME, Melniczek JR, et al. Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs. Mol Ther 2002; 5: 141-153.
22. Xu L, Mango RL, Sands MS, Haskins ME, Ellinwood NM, Ponder KP. Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Mol Ther 2002; 6: 745-758.
23. Ponder KP, Melniczek JR, Xu L, et al. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc Natl Acad Sci USA 2002; 99: 13102-13107.
24. Mah C, Cresawn KO, Fraites TJ Jr, et al. Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors. Gene Ther 2005; 12: 1405-1409. (Pubitemid 43090390)
25. VandenDriessche T, Vanslembrouck V, Goovaerts I, et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci USA 1999; 96: 10379-10384.
26. Xu L, Gao C, Sands MS, et al. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood 2003; 101: 3924-3932.
27. Zhang J, Xu L, Haskins ME, Parker Ponder K. Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs. Blood 2004; 103: 143-151. (Pubitemid 38029930)
28. Nakai H, Yant SR, Storm TA, Fuess S, Meuse L, Kay MA. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 2001; 75: 6969-6976.
29. Malik P, McQuiston SA, Yu XJ, 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 1997; 71: 1776-1783.
30. Flotte TR. Adeno-associated virus-based gene therapy for inherited disorders. Pediatr Res 2005; 58: 1143-1147. (Pubitemid 43755688)
31. Grimm D, Pandey K, Nakai H, Storm TA, Kay MA. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol 2006; 80: 426-439.
32. Conlon TJ, Cossette T, Erger K, et al. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther 2005; 12: 867-875.
33. Parsons TJ, Power C, Manor O. Fetal and early life growth and body mass index from birth to early adulthood in 1958 British cohort: longitudinal study. BMJ 2001; 323: 1331-1335.
34. Nelson WE. Growth and development in the infant and child. In: Textbook of Pediatrics, 8th edn, Nelson WE (ed.). WB Saunders Company: Philadelphia, PA, 1964; 21.
35. Nelson WE. Growth and development in the infant and child. In: Textbook of Pediatrics, 8th edn, Nelson WE (ed.). WB Saunders Company: Philadelphia, PA, 1964; 24.
36. Stocker JT, Dehner LP. Pediatric Pathology, Lippincott, Williams and Wilkins: Philadephia, PA, 2002.
37. Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 2008; 118: 3132-3142.
38. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419.
39. Wang L, Wang H, Bell P, et al. Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther 2010; 18: 118-125.
40. Snyder RO,Miao CH, Patijn GA, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997; 16: 270-276.
41. Snyder RO, Spratt SK, Lagarde C, et al. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther 1997; 8: 1891-1900.
42. Kessler PD, Podsakoff GM, Chen X, et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 1996; 93: 14082-14087.
43. Thomas CE, Storm TA, Huang Z, Kay MA. Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors. J Virol 2004; 78: 3110-3122. (Pubitemid 38314369)
44. Sipo I, Fechner H, Pinkert S, et al. Differential internalization and nuclear uncoating of self-complementary adeno-associated virus pseudotype vectors as determinants of cardiac cell transduction. Gene Ther 2007; 14: 1319-1329.
45. Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 9, and Rh10 in the mouse brain. Mol Ther 2006; 13: 528-537.
46. Lebherz C, Gao G, Louboutin JP, Millar J, Rader D, Wilson JM. Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia. J Gene Med 2004; 6: 663-672.
47. Gao GP, Lu Y, Sun X, et al. High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol 2006; 80: 6192-6194. (Pubitemid 43849199)
48. Gao GP, AlviraMR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 2002; 99: 11854-11859.
49. Blankinship MJ, Gregorevic P, Allen JM, et al. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 2004; 10: 671-678.
50. Bish LT, Morine K, Sleeper MM, et al. Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. Hum Gene Ther 2008; 19: 1359-1368.
51. Fausto N, Campbell JS. The role of hepatocytes and oval cells in liver regeneration and repopulation. Mech Dev 2003; 120: 117-130. (Pubitemid 35462100)
52. Magami Y, Azuma T, Inokuchi H, et al. Cell proliferation and renewal of normal hepatocytes and bile duct cells in adult mouse liver. Liver 2002; 22: 419-425.
53. Miao CH, Snyder RO, Schowalter DB, et al. The kinetics of rAAV integration in the liver. Nat Genet 1998; 19: 13-15.
54. Song S, Lu Y, Choi YK, et al. DNA-dependent PK inhibits adeno-associated virus DNA integration. Proc Natl Acad Sci USA 2004; 101: 2112-2126.
55. Gau CL, Rosenblatt RA, Cerullo V, et al. Short-term correction of arginase deficiency in a neonatal murine model with a helper-dependent adenoviral vector. Mol Ther 2009; 17: 1155-1163.