Preexisting Neutralizing Antibodies to Adeno-Associated Virus Capsids in Large Animals Other Than Monkeys May Confound in Vivo Gene Therapy Studies

Human Gene Therapy Methods

Volumn 26, Issue 3, 2015, Pages 103-105

  Calcedo, Roberto ^a   Franco, Judith ^a   Qin, Qiuyue ^a   Richardson, Dean W ^b   Mason, Jeffery B ^b   Boyd, Surina ^a   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 1; ADENO ASSOCIATED VIRUS 2; ADENO ASSOCIATED VIRUS 5; ADENO ASSOCIATED VIRUS 6; ADENO ASSOCIATED VIRUS 8; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY DETECTION; ARTICLE; CONTROLLED STUDY; EXPERIMENTAL DOG; EXPERIMENTAL HORSE; EXPERIMENTAL PIG; IMMUNOASSAY; IN VITRO STUDY; IN VIVO GENE TRANSFER; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; SEROTYPE; VIRAL GENE DELIVERY SYSTEM; VIRUS CAPSID; ADVERSE EFFECTS; ANIMAL; C57BL MOUSE; DEPENDOPARVOVIRUS; DOG; GENE THERAPY; GENETICS; HORSE; IMMUNOLOGY; PIG; PROCEDURES; VETERINARY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS; EQUIDAE; MIRIDAE; MUS; SUIDAE;

ANIMALS; ANTIBODIES, NEUTRALIZING; CAPSID; DEPENDOVIRUS; DOGS; GENETIC THERAPY; HORSES; MICE; MICE, INBRED C57BL; SWINE;

EID: 84932605736     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2015.082     Document Type: Article

References (14)

1. Scallan CD, Jiang H, Liu T, et al. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood 2006;107:1810-1817.
2. Hurlbut GD, Ziegler RJ, Nietupski JB, et al. Preexisting immunity and low expression in primates highlight translational challenges for liver-directed AAV8-mediated gene therapy. Mol Ther 18:1983-1994.
3. Jiang H, Couto LB, Patarroyo-White S, et al. Effects of transient immunosuppression on adenoassociated, virusmediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 2006;108: 3321-3328.
4. Wang L, Calcedo R, Bell P, Lin J, Grant RL, Siegel DL, Wilson JM. Impact of pre-existing immunity of gene transfer to nonhuman primate liver with AAV8. Hum Gen Ther 2011;22:1389-1401.
5. Frisbie DD, McIlwraith CW. Evaluation of gene therapy as a treatment for equine traumatic arthritis and osteoarthritis. Clin Orthop Relat Res 2000(379 Suppl):S273-287.
6. Wang Z, Kuhr CS, Allen JM, et al. Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression. Mol Ther 2007;15:1160-1166.
7. Yuasa K, Yoshimura M, Urasawa N, et al. Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products. Gene Ther 2007;14:1249-1260.
8. Wang L, Nichols TC, Read MS, et al. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol Ther 2000;1: 154-158.
9. Tao Z, Chen B, Tan X, et al. Coexpression of VEGF and angiopoietin-1 promotes angiogenesis and cardiomyocyte proliferation reduces apoptosis in porcine myocardial infarction (MI) heart. Proc Natl Acad Sci U S A 108:2064-2069.
10. Hadri L, Bobe R, Kawase Y, et al. SERCA2a gene transfer enhances eNOS expression and activity in endothelial cells. Mol Ther 18:1284-1292.
11. Gao G, Lu Y, Calcedo R, et al. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol Ther 2006;13:77-87.
12. Wang L, Calcedo R, Wang H, et al. The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 18:126-134.
13. Calcedo R, Vandenberghe LH, Gao G, et al. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 2009;199:381-390.
14. Hakim CH, Yue Y, Shin JH, et al. Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs. Mol Ther Methods Clin Dev 2014;1:14002.