Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia

Human Gene Therapy

Volumn 23, Issue 4, 2011, Pages 407-418

  Demaster, Amanda ^a,b   Luo, Xiaoyan ^a   Curtis, Sarah ^a   Williams, Kyha D ^a   Landau, Dustin J ^a   Drake, Elizabeth J ^a   Kozink, Daniel M ^a,c   Bird, Andrew ^a   Crane, Bayley ^c   Sun, Francis ^a   Pinto, Carlos R ^c,d   Brown, Talmage T ^c   Kemper, Alex R ^a   Koeberl, Dwight D ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b East Lincoln Animal Hospital   (United States)

^c NORTH CAROLINA STATE UNIVERSITY   (United States)

^d OHIO STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ASPARTATE AMINOTRANSFERASE; GLUCOSE 6 PHOSPHATASE; GLYCOGEN; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 2; ADENO ASSOCIATED VIRUS 9; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CONTROLLED STUDY; DIET RESTRICTION; DOG; DRUG EFFICACY; ENZYME ACTIVITY; FEMALE; GLYCEMIC CONTROL; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 1A; HYPOGLYCEMIA; INFLAMMATION; MALE; MORTALITY; NEWBORN; NONHUMAN; PSEUDOTYPING; RECURRENCE RISK; SURVIVAL; TREATMENT OUTCOME; VIRAL GENE THERAPY; VIRUS GENOME;

ANIMALS; DEPENDOVIRUS; DOGS; GENETIC THERAPY; GENETIC VECTORS; GLUCOSE-6-PHOSPHATASE; GLYCOGEN STORAGE DISEASE TYPE I; HYPOGLYCEMIA; LIVER;

EID: 84860155178     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.106     Document Type: Article

References (30)

1. Beaty, R. M., Jackson, M., Peterson, D., et al. (2002). Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors. Gene Ther. 9, 1015-1022.
2. Brix, A. E., Howerth, E. W., McConkie-Rosell, A., et al. (1995). Glycogen storage disease type Ia in two littermate Maltese puppies. Vet. Pathol. 32, 460-465.
3. Chen, Y. T. (2001). Glycogen storage diseases. In The Metabolic and Molecular Bases of Inherited Disease. C. R. Scriver, A. L., Beaudet, W. S. Sly, and D. Valle, eds. (McGraw-Hill, New York) pp. 1521-1551.
4. Chen, Y. T., Cornblath, M., and Sidbury, J. B. (1984). Cornstarch therapy in type I glycogen-storage disease. N. Engl. J. Med. 310, 171-175. (Pubitemid 14224111)
5. Chen, Y. T., Bazzarre, C. H., Lee, M. M., et al. (1993). Type I glycogen storage disease: nine years of management with cornstarch. Eur. J. Pediatr. 152 Suppl. 1, S56-S59. (Pubitemid 23134780)
6. Crane, B., Luo, X., Demaster, A., et al. (2011). Rescue administration of a helper-dependent adenovirus vector with longterm efficacy in dogs with glycogen storage disease type Ia. Gene. Ther. Epub ahead of print.
7. Gao, G. P., Alvira, M. R., Wang, L., et al. (2002). Novel adenoassociated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U. S. A. 99, 11854-11859. (Pubitemid 34994488)
8. Halbert, C. L., Standaert, T. A., Aitken, M. L., et al. (1997). Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. Virology 71, 5932-5941. (Pubitemid 27304930)
9. Halbert, C. L., Rutledge, E. A., Allen, J. M., et al. (2000). Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. Virology 74, 1524-1532. (Pubitemid 30044103)
10. Herzog, R. W., Davidoff, A. M., Markusic, D. M., and Nathwani, A. C. (2011). AAV vector biology in primates: finding the missing link? Mol. Ther. 19, 1923-1924.
11. Holmlund, E., Nohynek, H., Quiambao, B., et al. (2011). Motherinfant vaccination with pneumococcal polysaccharide vaccine: persistence of maternal antibodies and responses of infants to vaccination. Vaccine Epub ahead of print.
12. Keller, K. M., Schutz, M., Podskarbi, T., et al. (1998). A new mutation of the glucose-6-phosphatase gene in a 4-year-old girl with oligosymptomatic glycogen storage disease type 1a. J. Pediatr. 132, 360-361. (Pubitemid 28125198)
13. Kikuchi, M., Hasegawa, K., Handa, I., et al. (1991). Chronic pancreatitis in a child with glycogen storage disease type 1. Eur. J. Pediatr. 150, 852-853.
14. Kishnani, P. S., Faulkner, E., Vancamp, S., et al. (2001). Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia). Vet. Pathol. 38, 83-91. (Pubitemid 33621676)
15. Koeberl, D. D., Sun, B. D., Damodaran, T. V., et al. (2006). Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. Gene Ther. 13, 1281-1289. (Pubitemid 44264082)
16. Koeberl, D. D., Pinto, C., Sun, B., et al. (2008). AAV vectormediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia. Mol. Ther. 16, 665-672. (Pubitemid 351426167)
17. Koeberl, D. D., Kishnani, P. S., Bali, D., and Chen, Y. T. (2009). Emerging therapies for glycogen storage disease type I. Trends Endocrinol. Metab. 20, 252-258.
18. Leuridan, E., and Van Damme, P. (2007). Passive transmission and persistence of naturally acquired or vaccine-induced maternal antibodies against measles in newborns. Vaccine 25, 6296-6304. (Pubitemid 47090234)
19. Luo, X., Hall, G., Li, S., et al. (2011). Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector. Mol. Ther. Epub ahead of print.
20. Manno, C. S., Pierce, G. F., Arruda, V. R., 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)
21. Matern, D., Starzl, T. E., Arnaout, W., et al. (1999). Liver transplantation for glycogen storage disease types I, I. I. I, and IV. Eur. J. Pediatr. 158 Suppl. 2, S43-S48.
22. Snyder, R. O., and Francis, J. (2005). Adeno-associated viral vectors for clinical gene transfer studies. Curr. Gene Ther. 5, 311-321. (Pubitemid 40895015)
23. Sun, B., Li, S., Yang, L., et al. (2009). Activation of glycolysis and apoptosis in glycogen storage disease type Ia. Mol. Genet. Metab. 97, 267-271.
24. Sun, M. S., Pan, C. J., Shieh, J. J., et al. (2002). Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice. Hum. Mol. Genet. 11, 2155-2164.
25. Wang, L., Calcedo, R., Nichols, T. C., 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.
26. Wang, L., Bell, P., Lin, J., et al. (2011a). AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol. Ther. 19, 2012-2020.
27. Wang, L., Calcedo, R., Bell, P., et al. (2011b). Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors. Hum. Gene Ther. Epub ahead of print.
28. Weinstein, D. A., Correia, C. E., Conlon, T., et al. (2010). Adenoassociated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum Gene. Ther. 21, 903-910.
29. Wolfsdorf, J. I., and Crigler, J. F., Jr. (1999). Effect of continuous glucose therapy begun in infancy on the long-term clinical course of patients with type I glycogen storage disease. J. Pediatr. Gastroenterol. Nutr. 29, 136-143. (Pubitemid 30366162)
30. Yiu, W. H., Lee, Y. M., Peng, W. T., et al. (2010). Complete normalization of hepatic G6pc deficiency in murine glycogen storage disease type Ia using gene therapy. Mol. Ther. 18, 1076-1084.