Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer

Molecular Therapy

Volumn 19, Issue 5, 2011, Pages 870-875

  Salido, Eduardo ^a,c   Rodriguez Pena, Marisol ^b   Santana, Alfredo ^a   Beattie, Stuart G ^b   Petry, Harald ^b   Torres, Armando ^a  

^a UNIVERSIDAD DE LA LAGUNA   (Spain)

^b Amsterdam Molecular Therapeutics ^*  (Netherlands)

^c HOSPITAL UNIVERSITARIO DE CANARIAS   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE GLYOXYLATE AMINOTRANSFERASE; COMPLEMENTARY DNA; ETHYLENE GLYCOL; GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 5; ADENO ASSOCIATED VIRUS SEROTYPE 8; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EXCRETION; DRUG MEGADOSE; ENZYME ACTIVITY; FEMALE; GENE TRANSFER; HYPEROXALURIA; IMMUNOGENICITY; KIDNEY CALCIFICATION; KIDNEY FAILURE; LIVER TOXICITY; LOW DRUG DOSE; MALE; MORTALITY; MOUSE; MULTIPLE CYCLE TREATMENT; NONHUMAN; OXALOSIS 1; PHENOTYPE; PSEUDOTYPING; TREATMENT DURATION; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID; VIRUS RECOMBINANT;

ADENO-ASSOCIATED VIRUS; MIRIDAE; MUS;

EID: 79955581947     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.270     Document Type: Article

References (32)

1. Verma, IM and Weitzman, MD (2005). Gene therapy: twenty-first century medicine. Annu Rev Biochem 74: 711-738. (Pubitemid 40995522)
2. Arruda, VR, Stedman, HH, Nichols, TC, Haskins, ME, Nicholson, M, Herzog, RW et al. (2005). Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves longterm correction of hemophilia B in a large animal model. Blood 105: 3458-3464. (Pubitemid 40628186)
3. Glatzel, M, Flechsig, E, Navarro, B, Klein, MA, Paterna, JC, Büeler, H et al. (2000). Adenoviral and adeno-associated viral transfer of genes to the peripheral nervous system. Proc Natl Acad Sci USA 97: 442-447. (Pubitemid 30055850)
4. Gregorevic, P, Blankinship, MJ, Allen, JM, Crawford, RW, Meuse, L, Miller, DG et al. (2004). Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med 10: 828-834. (Pubitemid 39070856)
5. Grimm, D, Zhou, S, Nakai, H, Thomas, CE, Storm, TA, Fuess, S et al. (2003). Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 102: 2412-2419. (Pubitemid 37193577)
6. Paterna, JC, Feldon, J and Büeler, H (2004). Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats. J Virol 78: 6808-6817. (Pubitemid 38781518)
7. Sarkar, R, Tetreault, R, Gao, G, Wang, L, Bell, P, Chandler, R et al. (2004). Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood 103: 1253-1260. (Pubitemid 38168635)
8. Flotte, TR, Zeitlin, PL, Reynolds, TC, Heald, AE, Pedersen, P, Beck, S et al. (2003). Phase I trial of intranasal and endobronchial administration of a recombinant adenoassociated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther 14: 1079-1088. (Pubitemid 36851399)
9. Kay, MA, Manno, CS, Ragni, MV, Larson, PJ, Couto, LB, McClelland, A et al. (2000). Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24: 257-261. (Pubitemid 30132193)
10. Manno, CS, Chew, AJ, Hutchison, S, Larson, PJ, Herzog, RW, Arruda, VR et al. (2003). AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101: 2963-2972. (Pubitemid 36857981)
11. Wagner, JA, Nepomuceno, IB, Messner, AH, Moran, ML, Batson, EP, Dimiceli, S et al. (2002). 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: 1349-1359. (Pubitemid 35034514)
12. Manno, CS, Pierce, GF, Arruda, VR, Glader, B, Ragni, M, Rasko, JJ et al. (2006). Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12: 342-347. (Pubitemid 43355084)
13. Brantly, ML, Chulay, JD, Wang, L, Mueller, C, Humphries, M, Spencer, LT et al. (2009). Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci USA 106: 16363-16368.
14. McKeon, C and Samulski, RJ (1996). NIDDK Workshop on AAV Vectors: Gene Transfer into Quiescent Cells. Hum Gene Ther 7: 1615-1619. (Pubitemid 26302216)
15. Nakai, H, Thomas, CE, Storm, TA, Fuess, S, Powell, S, Wright, JF et al. (2002). A limited number of transducible hepatocytes restricts a wide-range linear vector dose-response in recombinant adeno-associated virus-mediated liver transduction. J Virol 76: 11343-11349. (Pubitemid 35231289)
16. Gao, GP, Alvira, MR, Wang, L, Calcedo, R, Johnston, J and Wilson, JM (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859. (Pubitemid 34994488)
17. Wu, Z, Asokan, A and Samulski, RJ (2006). Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14: 316-327. (Pubitemid 44184972)
18. Büning, H, Perabo, L, Coutelle, O, Quadt-Humme, S and Hallek, M (2008). Recent developments in adeno-associated virus vector technology. J Gene Med 10: 717-733. (Pubitemid 352003915)
19. Danpure, CJ (1995). Advances in the enzymology and molecular genetics of primary hyperoxaluria type 1. Prospects for gene therapy. Nephrol Dial Transplant 10 Suppl 8: 24-29.
20. Gao, G, Vandenberghe, LH and Wilson, JM (2005). New recombinant serotypes of AAV vectors. Curr Gene Ther 5: 285-297. (Pubitemid 40895013)
21. Boutin, S, Monteilhet, V, Veron, P, Leborgne, C, Benveniste, O, Montus, MF et al. (2010). Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum Gene Ther 21: 704-712.
22. Salido, EC, Li, XM, Lu, Y, Wang, X, Santana, A, Roy-Chowdhury, N et al. (2006). Alanine-glyoxylate aminotransferase-deficient mice, a model for primary hyperoxaluria that responds to adenoviral gene transfer. Proc Natl Acad Sci USA 103: 18249-18254. (Pubitemid 44871644)
23. Kramer, MG, Barajas, M, Razquin, N, Berraondo, P, Rodrigo, M, Wu, C et al. (2003). In vitro and in vivo comparative study of chimeric liver-specific promoters. Mol Ther 7: 375-385. (Pubitemid 36503861)
24. Nakai, H, Fuess, S, Storm, TA, Muramatsu, S, Nara, Y and Kay, MA (2005). Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol 79: 214-224. (Pubitemid 39650967)
25. Bell, P, Wang, L, Lebherz, C, Flieder, DB, Bove, MS, Wu, D et al. (2005). No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. Mol Ther 12: 299-306. (Pubitemid 41025100)
26. Nathwani, AC, Gray, JT, McIntosh, J, Ng, CY, Zhou, J, Spence, Y et al. (2007). Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood 109: 1414-1421. (Pubitemid 46239572)
27. Davidoff, AM, Ng, CY, Zhou, J, Spence, Y and Nathwani, AC (2003). Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 102: 480-488. (Pubitemid 36841964)
28. Pañeda, A, Vanrell, L, Mauleon, I, Crettaz, JS, Berraondo, P, Timmermans, EJ et al. (2009). Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders. Hum Gene Ther 20: 908-917.
29. Gao, G, Wang, Q, Calcedo, R, Mays, L, Bell, P, Wang, L et al. (2009). Adeno-associated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther 20: 930-942.
30. Wang, L, Wang, H, Bell, P, McCarter, RJ, He, J, Calcedo, R et al. (2010). Systematic evaluation of AAV vectors for liver directed gene transfer in murine models. Mol Ther 18: 118-125.
31. Yasue, T (1969). Histochemical identification of calcium oxalate. Acta Histochem Cytochem 2: 83-95.
32. Hermens, WT, ter Brake, O, Dijkhuizen, PA, Sonnemans, MA, Grimm, D, Kleinschmidt, JA et al. (1999). Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system. Hum Gene Ther 10: 1885-1891. (Pubitemid 29357694)