Preferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes

Molecular Genetics and Metabolism Reports

Volumn 6, Issue , 2016, Pages 48-54

  Gilkes, J A ^a   Bloom, M D ^a   Heldermon, C D ^a  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

AAV; Adeno associated virus; Gene therapy; Lysosomal storage disease; MPS IIIB; Mucopolysaccharidosis type III B

Indexed keywords

ACETYLGLUCOSAMINIDASE;

ADENO ASSOCIATED VIRUS 5; ADENO ASSOCIATED VIRUS 8; ADENO ASSOCIATED VIRUS 9; AMYGDALOID NUCLEUS; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; EPENDYMA CELL; FEASIBILITY STUDY; GENOTYPE; HIPPOCAMPUS; HYPOTHALAMUS; MOUSE; NONHUMAN; SANFILIPPO SYNDROME; SANFILIPPO SYNDROME TYPE B; THALAMUS; VIRAL GENE THERAPY;

EID: 84957438385     PISSN: None     EISSN: 22144269     Source Type: Journal    
DOI: 10.1016/j.ymgmr.2015.11.006     Document Type: Article

References (28)

1. G. Yogalingam, and J.J. Hopwood Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: diagnostic, clinical, and biological implications Hum. Mutat. 18 2001 264 281
2. J.A. Gilkes, and C.D. Heldermon Mucopolysaccharidosis III (Sanfilippo syndrome) - disease presentation and experimental therapies Pediatr. Endocrinol. Rev. 12 Suppl. 1 2014 133 140
3. H.H. Li, and et al. Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding ?-N-acetylglucosaminidase Proc. Natl. Acad. Sci. U. S. A. 96 1999 14505 14510
4. C.D. Heldermon, and et al. Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo Syndrome Type B PLoS One 2 2007 e772
5. T.R. Flotte Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors Gene Ther. 11 2004 805 810
6. M.G. Kaplitt, and et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain Nat. Genet. 8 1994 148 154
7. F. Mingozzi, and K.A. High Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges Nat Rev Genet 12 2011 341 355
8. J.E. Rabinowitz, and et al. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity J. Virol. 76 2002 791 801
9. G. Gao, and et al. Clades of adeno-associated viruses are widely disseminated in human tissues J. Virol. 78 2004 6381 6388
10. J.E. Rabinowitz, and et al. Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups J. Virol. 78 2004 4421 4432
11. C. Burger, and et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system Mol. Ther. 10 2004 302 317
12. J.-M. Taymans, and et al. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain Hum. Gene Ther. 18 2007 195 206
13. P. Chakrabarty, and et al. Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain PLoS One 8 2013 e67680
14. T.M. Daly, C. Vogler, B. Levy, M.E. Haskins, and M.S. Sands Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease Proc. Natl. Acad. Sci. U. S. A. 96 1999 2296 2300
15. M.L.D. Broekman, L.A. Comer, B.T. Hyman, and M. Sena-Esteves Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain Neuroscience 138 2006 501 510
16. B.L. Gliddon, and J.J. Hopwood Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice Pediatr. Res. 56 2004 65 72
17. C.D. Heldermon, and et al. Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice Gene Ther. 20 2013 913 921
18. C.D. Heldermon, and et al. Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB Mol. Ther. 18 2010 873 880
19. M. Nonnenmacher, and T. Weber Intracellular transport of recombinant adeno-associated virus vectors Gene Ther. 19 2012 649 658
20. C.N. Cearley, and J.H. Wolfe Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain Mol. Ther. 13 2006 528 537
21. R. Holehonnur, and et al. Adeno-associated viral serotypes produce differing titers and differentially transduce neurons within the rat basal and lateral amygdala BMC Neurosci. 15 2014 28
22. B.L. Davidson, and et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system Proc. Natl. Acad. Sci. 97 2000 3428 3432
23. A.F. Skorupa, K.J. Fisher, J.M. Wilson, M.K. Parente, and J.H. Wolfe Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice Exp. Neurol. 160 1999 17 27
24. N.U. Barua, and et al. Convection-enhanced delivery of AAV2 in white matter - a novel method for gene delivery to cerebral cortex J. Neurosci. Methods 220 2013 1 8
25. A.P. Kells, and et al. Efficient gene therapy-based method for the delivery of therapeutics to primate cortex PNAS 106 2009 2407 2411
26. N. Carty, and et al. Convection-enhanced delivery and systemic mannitol increase gene product distribution of AAV vectors 5, 8, and 9 and increase gene product in the adult mouse brain J. Neurosci. Methods 194 2010 144 153
27. S. Zolotukhin, and et al. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors Methods 28 2002 158 167
28. J. Marsh, and A.H. Fensom 4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease Clin. Genet. 27 1985 258 262