Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia

Gene Therapy

Volumn 19, Issue 4, 2012, Pages 443-452

  Crane, B ^a,b   Luo, X ^b,e   Demaster, A ^b   Williams, K D ^b   Kozink, D M ^a   Zhang, P ^b   Brown, T T ^a   Pinto, C R ^a   Oka, K ^c   Sun, F ^b   Jackson, M W ^d   Chan, L ^c   Koeberl, D D ^b  

^a NORTH CAROLINA STATE UNIVERSITY   (United States)

^b DUKE UNIVERSITY MEDICAL CENTER   (United States)

^c BAYLOR COLLEGE OF MEDICINE   (United States)

^d UNIVERSITY OF GLASGOW   (United Kingdom)

^e OHIO STATE UNIVERSITY   (United States)

Author keywords

adenovirus vector; glucose 6 phosphatase; glycogen storage disease; renal dysfunction; von Gierke disease

Indexed keywords

ADENOVIRUS VECTOR; GLUCOSE; GLUCOSE 6 PHOSPHATASE; GLYCOGEN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANOREXIA; ARTICLE; CONTROLLED STUDY; GENE THERAPY; GLUCOSE BLOOD LEVEL; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 1A; HYPOGLYCEMIA; KIDNEY DYSFUNCTION; LETHARGY; LIVER BIOPSY; NONHUMAN; PRIORITY JOURNAL; SURVIVAL;

ADENOVIRIDAE; ANIMALS; BODY WEIGHT; DOG DISEASES; DOGS; GENE THERAPY; GENETIC VECTORS; GLUCOSE-6-PHOSPHATASE; GLYCOGEN STORAGE DISEASE TYPE I; HYPOGLYCEMIA;

ADENOVIRIDAE; CANIS FAMILIARIS;

EID: 84859592442     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.86     Document Type: Article

References (32)

1. Chen YT. In: CR Scriver, AL Beaudet, WS Sly, D Valle (eds). The Metabolic and Molecular Bases of Inherited Disease. McGraw-Hill: New York, 2001, pp 1521-1551.
2. Chou JY, Matern D, Mansfield BC, Chen YT. Type I glycogen storage diseases: disorders of the glucose-6-phosphatase complex. Curr Mol Med 2002; 2: 121-143. (Pubitemid 34649836)
3. Brix AE, Howerth EW, McConkie-Rosell A, Peterson D, Egnor D, Wells MR et al. Glycogen storage disease type Ia in two littermate Maltese puppies. Vet Pathol 1995; 32: 460-465.
4. Kishnani PS, Faulkner E, VanCamp S, Jackson M, Brown T, Boney A et al. Canine model and genomic structural organization of glycogen storage disease type Ia (GSD Ia). Vet Pathol 2001; 38: 83-91. (Pubitemid 33621676)
5. Williams JC. Nutritional goals in glycogen storage disease. N Engl J Med 1986; 314: 709-710. (Pubitemid 16164104)
6. Chou JY, Mansfield BC. Gene therapy for type I glycogen storage diseases. Curr Gene Ther 2007; 7: 79-88. (Pubitemid 46681766)
7. Beaty RM, Jackson M, Peterson D, Bird A, Brown T, Benjamin Jr DK et al. Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors. Gene Therapy 2002; 9: 1015-1022.
8. Koeberl DD, Pinto C, Sun B, Li S, Kozink DM, Benjamin Jr DK et al. AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia. Mol Ther 2008; 16: 665-672. (Pubitemid 351426167)
9. Weinstein DA, Correia CE, Conlon T, Specht A, Verstegen J, Onclin-Verstegen K et al. Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia. Hum Gene Ther 2010; 21: 903-910.
10. Brunetti-Pierri N, Ng P. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Therapy 2008; 15: 553-560.
11. Jozkowicz A, Dulak J. Helper-dependent adenoviral vectors in experimental gene therapy. Acta Biochim Pol 2005; 52: 589-599. (Pubitemid 41571704)
12. Morral N, Parks RJ, Zhou H, Langston C, Schiedner G, Quinones J et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity. Hum Gene Ther 1998; 9: 2709-2716. (Pubitemid 29027014)
13. Brunetti-Pierri N, Ng T, Iannitti DA, Palmer DJ, Beaudet AL, Finegold MJ et al. Improved hepatic transduction, reduced systemic vector dissemination, and longterm transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates. Hum Gene Ther 2006; 17: 391-404.
14. Brunetti-Pierri N, Stapleton GE, Law M, Breinholt J, Palmer DJ, Zuo Y et al. Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther 2009; 17: 327-333.
15. Kim IH, Jozkowicz A, Piedra PA, Oka K, Chan L. Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 2001; 98: 13282-13287. (Pubitemid 33051354)
16. McCormack Jr WM, Seiler MP, Bertin TK, Ubhayakar K, Palmer DJ, Ng P et al. Helper-dependent adenoviral gene therapy mediates long-term correction of the clotting defect in the canine hemophilia A model. J Thromb Haemost 2006; 4: 1218-1225.
17. Wolfsdorf JI, Laffel LM, Crigler Jr JF. Metabolic control and renal dysfunction in type I glycogen storage disease. J Inher Metab Dis 1997; 20: 559-568. (Pubitemid 27354852)
18. Kishnani PS, Bao Y, Wu JY, Brix AE, Lin JL, Chen YT. Isolation and nucleotide sequence of canine glucose-6-phosphatase mRNA: identification of mutation in puppies with glycogen storage disease type Ia. Biochem Mol Med 1997; 61: 168-177. (Pubitemid 27347220)
19. Chen YT. Type I glycogen storage disease: kidney involvement, pathogenesis and its treatment. Pediatr Nephrol 1991; 5: 71-76.
20. Chen YT, Coleman RA, Scheinman JI, Kolbeck PC, Sidbury JB. Renal disease in type I glycogen storage disease. New Engl J Med 1988; 318: 7-11. (Pubitemid 18049531)
21. Yiu WH, Lee YM, Peng WT, Pan CJ, Mead PA, Mansfield BC et al. Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy. Mol Ther 2010; 18: 1076-1084.
22. Koeberl DD, Sun B, Bird A, Chen Y, Oka K, Chan L. Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia. Mol Ther 2007; 15: 1253-1258. (Pubitemid 46965320)
23. Matern D, Starzl TE, Arnaout W, Barnard J, Bynon JS, Dhawan A et al. Liver transplantation for glycogen storage disease types I, III, and IV and. Eur J Pediatr 1999; 158 (Suppl 2): S43-S48.
24. Wolfsdorf JI, Crigler Jr JF. Cornstarch regimens for nocturnal treatment of young adults with type I glycogen storage disease. Am J Clin Nutr 1997; 65: 1507-1511. (Pubitemid 27187765)
25. Hu C, Cela RG, Suzuki M, Lee B, Lipshutz GS. Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc Natl Acad Sci USA 2011; 108: 2082-2087.
26. Koeberl DD, Sun BD, Damodaran TV, Brown T, Millington DS, Benjamin DJ et al. Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. Gene Therapy 2006; 13: 1281-1289. (Pubitemid 44264082)
27. Reddy SK, Kishnani PS, Sullivan JA, Koeberl DD, Desai DM, Skinner MA et al. Resection of hepatocellular adenoma in patients with glycogen storage disease type Ia. J Hepatol 2007; 47: 658-663. (Pubitemid 47503424)
28. Yiu WH, Pan CJ, Mead PA, Starost MF, Mansfield BC, Chou JY. Normoglycemia alone is insufficient to prevent long-term complications of hepatocellular adenoma in glycogen storage disease type Ib mice. J Hepatol 2009; 51: 909-917.
29. Oka K, Pastore L, Kim IH, Merched A, Nomura S, Lee HJ et al. Long-term stable correction of low-density lipoprotein receptor-deficient mice with a helper-dependent adenoviral vector expressing the very low-density lipoprotein receptor. Circulation 2001; 103: 1274-1281. (Pubitemid 32221352)
30. Nomura S, Merched A, Nour E, Dieker C, Oka K, Chan L. Low-density lipoprotein receptor gene therapy using helper-dependent adenovirus produces long-term protection against atherosclerosis in a mouse model of familial hypercholesterolemia. Gene Therapy 2004; 11: 1540-1548. (Pubitemid 39359462)
31. Parks RJ, Chen L, Anton M, Sankar U, Rudnicki MA, Graham FL. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc Natl Acad Sci USA 1996; 93: 13565-13570. (Pubitemid 26424159)
32. Parks R, Evelegh C, Graham F. Use of helper-dependent adenoviral vectors of alternative serotypes permits repeat vector administration. Gene Therapy 1999; 6: 1565-1573. (Pubitemid 29465515)