AAV8-mediated hepatic gene transfer in infant rhesus monkeys (macaca mulatta)

Molecular Therapy

Volumn 19, Issue 11, 2011, Pages 2012-2020

  Wang, Lili ^a   Bell, Peter ^a   Lin, Jianping ^a   Calcedo, Roberto ^a   Tarantal, Alice F ^b,c   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

ADOLESCENT; AGE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; CONTROLLED STUDY; GENE EXPRESSION; LIVER; LIVER CELL; MACACA MULATTA; MALE; NONHUMAN; PERINATAL PERIOD; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS; FELIDAE; MACACA MULATTA; MIRIDAE; PRIMATES;

EID: 80455174705     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.151     Document Type: Article

References (36)

1. Ponder, KP (2011). Hemophilia gene therapy: a Holy Grail found. Mol Ther 19: 427-428.
2. Graham, T, McIntosh, J, Work, LM, Nathwani, A and Baker, AH (2008). Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats. Genet Vaccines Ther 6: 9.
3. Cunningham, SC, Spinoulas, A, Carpenter, KH, Wilcken, B, Kuchel, PW and Alexander, IE (2009). AAV2/8-mediated correction of OTC defciency is robust in adult but not neonatal Spf(ash) mice. Mol Ther 17: 1340-1346.
4. Cotugno, G, Annunziata, P, Tessitore, A, O'Malley, T, Capalbo, A, Faella, A et al. (2011). Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Mol Ther 19: 461-469.
5. Monahan, PE, Lothrop, CD, Sun, J, Hirsch, ML, Kafri, T, Kantor, B et al. (2010). Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: a strategy for broad clinical application. Mol Ther 18: 1907-1916.
6. Wang, L, Calcedo, R, Nichols, TC, Bellinger, DA, Dillow, A, Verma, IM et al. (2005). Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 105: 3079-3086.
7. Bell, P, Gao, G, Haskins, ME, Wang, L, Sleeper, M, Wang, H et al. (2011). Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Hum Gene Ther. (In press).
8. Nathwani, AC, Gray, JT, McIntosh, J, Ng, CY, Zhou, J, Spence, Y et al. (2007). Safe and effcient 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)
9. Nathwani, AC, Gray, JT, Ng, CY, Zhou, J, Spence, Y, Waddington, SN et al. (2006). Self-complementary adeno-associated virus vectors containing a novel liver-specifc human factor IX expression cassette enable highly effcient transduction of murine and nonhuman primate liver. Blood 107: 2653-2661.
10. Halbert, CL, Miller, AD, McNamara, S, Emerson, J, Gibson, RL, Ramsey, B et al. (2006). Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fbrosis and normal populations: Implications for gene therapy using AAV vectors. Hum Gene Ther 17: 440-447.
11. Nakai, H, Yant, SR, Storm, TA, Fuess, S, Meuse, L and Kay, MA (2001). Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 75: 6969-6976. (Pubitemid 32641584)
12. Wang, Z, Zhu, T, Qiao, C, Zhou, L, Wang, B, Zhang, J et al. (2005). Adeno-associated virus serotype 8 effciently delivers genes to muscle and heart. Nat Biotechnol 23: 321-328. (Pubitemid 41094420)
13. Cunningham, SC, Dane, A P, Spinoulas, A, Logan, GJ and Alexander, IE (2008). Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther 16: 1081-1088. (Pubitemid 351737073)
14. Hu, C, Busuttil, RW and Lipshutz, GS (2010). RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy. J Gene Med 12: 766-778.
15. Chandler, RJ and Venditti, CP (2010). Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy. Mol Ther 18: 11-16.
16. Carrillo-Carrasco, N, Chandler, RJ, Chandrasekaran, S and Venditti, CP (2010). Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Hum Gene Ther 21: 1147-1154.
17. Chandler, RJ, Chandrasekaran, S, Carrillo-Carrasco, N, Senac, JS, Hofherr, SE, Barry, MA et al. (2011). Adeno-associated virus serotype 8 gene transfer rescues a neonatal lethal murine model of propionic acidemia. Hum Gene Ther 22: 477-481.
18. Yiu, WH, Lee, YM, Peng, WT, Pan, CJ, Mead, PA, Mansfeld, BC et al. (2010). Complete normalization of hepatic G6PC defciency in murine glycogen storage disease type Ia using gene therapy. Mol Ther 18: 1076-1084.
19. Tessitore, A, Faella, A, O'Malley, T, Cotugno, G, Doria, M, Kunieda, T et al. (2008). Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle. Mol Ther 16: 30-37.
20. Weinstein, DA, Correia, CE, Conlon, T, Specht, A, Verstegen, J, Onclin-Verstegen, K et al. (2010). Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum Gene Ther 21: 903-910.
21. Jiang, H, Couto, LB, Patarroyo-White, S, Liu, T, Nagy, D, Vargas, JA et al. (2006). Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108: 3321-3328.
22. Hurlbut, GD, Ziegler, RJ, Nietupski, JB, Foley, JW, Woodworth, LA, Meyers, E et al. (2010). Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol Ther 18: 1983-1994.
23. Wang, L, Calcedo, R, Wang, H, Bell, P, Grant, R, Vandenberghe, LH et al. (2010). The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 18: 126-134.
24. Wang, L, Calcedo, R, Bell, P, Lin, J, Grant, RL, Siegel, DL et al. (2011). Impact of preexisting immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors impact of pre-existing immunity. Hum Gene Ther.(epub ahead of print)
25. Pacak, CA, Mah, CS, Thattaliyath, BD, Conlon, TJ, Lewis, MA, Cloutier, DE et al. (2006). Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ Res 99: e3-e9. (Pubitemid 44255502)
26. Tarantal, AF and Lee, CC (2010). Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta). Hum Gene Ther 21: 143-148.
27. Dane, A P, Cunningham, SC, Graf, NS and Alexander, IE (2009). Sexually dimorphic patterns of episomal rAAV genome persistence in the adult mouse liver and correlation with hepatocellular proliferation. Mol Ther 17: 1548-1554.
28. Lee, CC, Jimenez, DF, Kohn, DB and Tarantal, AF (2005). Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther 16: 417-425. (Pubitemid 40647139)
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-specifc T cell responses. Hum Gene Ther 20: 930-942.
30. Lock, M, Alvira, M, Vandenberghe, LH, Samanta, A, Toelen, J, Debyser, Z et al. (2010). Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther 21: 1259-1271.
31. Tarantal, AF (2005). Ultrasound imaging in rhesus (Macaca mulatta) and long-tailed (Macaca fascicularis) macaques: Reproductive and Research Applications. In: Wolfe-Coote, S (ed). The Laboratory Primate. Elsevier Academic Press: San Diego. pp 317-351.
32. Jimenez, DF and Tarantal, AF (2003). Fetal gender determination in early frst trimester pregnancies of rhesus monkeys (Macaca mulatta) by fuorescent PCR analysis of maternal serum. J Med Primatol 32: 315-319. (Pubitemid 38008521)
33. Tarantal, AF, McDonald, RJ, Jimenez, DF, Lee, CC, O'Shea, CE, Leapley, AC et al. (2005). Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and effciency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther 12: 87-98. (Pubitemid 40862360)
34. Calcedo, R, Vandenberghe, LH, Gao, G, Lin, J and Wilson, JM (2009). Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 199: 381-390.
35. Bell, P, Moscioni, AD, McCarter, RJ, Wu, D, Gao, G, Hoang, A et al. (2006). Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. Mol Ther 14: 34-44. (Pubitemid 43922140)
36. Vandenberghe, LH, Wang, L, Somanathan, S, Zhi, Y, Figueredo, J, Calcedo, R et al. (2006). Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 12: 967-971. (Pubitemid 44175151)