Inner limiting membrane barriers to aav-mediated retinal transduction from the vitreous

Molecular Therapy

Volumn 17, Issue 12, 2009, Pages 2096-2102

  Dalkara, Deniz ^a,b   Kolstad, Kathleen D ^a,c   Caporale, Natalia ^a,c   Visel, Meike ^a,c   Klimczak, Ryan R ^a,c   Schaffer, David V ^a,b   Flannery, John G ^a,c  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b University of California at Berkeley   (United States)

^c University of California Berkeley   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; PROTEINASE;

ADENO ASSOCIATED VIRUS; ADULT ANIMAL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ANTIBODY LABELING; ARTICLE; CELL POPULATION; CELL TYPE; CONTROLLED STUDY; ELECTRORETINOGRAPHY; EVOKED VISUAL RESPONSE; EYE PHOTOGRAPHY; FLUORESCENCE; GENETIC TRANSDUCTION; HISTOLOGY; MEMBRANE; NONHUMAN; RAT; RETINA; RETINA CELL; SEROTYPE; TRANSMISSION ELECTRON MICROSCOPY; VIRAL GENE THERAPY; VIRUS CAPSID; VIRUS PARTICLE; VISUAL CORTEX; VITREOUS BODY;

ANIMALS; CELL MEMBRANE; CELL MEMBRANE PERMEABILITY; DEPENDOVIRUS; ELECTRORETINOGRAPHY; FLUORESCEIN ANGIOGRAPHY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; PRONASE; RATS; RATS, SPRAGUE-DAWLEY; RETINA; TRANSDUCTION, GENETIC; VITREOUS BODY;

ADENO-ASSOCIATED VIRUS; MIRIDAE;

EID: 73849132937     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.181     Document Type: Article

References (39)

1. Mueller, C and Flotte, TR (2008). Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 15: 858-863.
2. Lebherz, C, Maguire, A, Tang, W, Bennett, J and Wilson, JM (2008). Novel AAV serotypes for improved ocular gene transfer. J Gene Med 10: 375-382.
3. Hauswirth, WW, Aleman, TS, Kaushal, S, Cideciyan, AV, Schwartz, SB, Wang, L et al. (2008). Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 19: 979-990.
4. Büning, H, Perabo, L, Coutelle, O, Quadt-Humme, S and Hallek, M (2008). Recent developments in adeno-associated virus vector technology. J Gene Med 10: 717-733.
5. Goncalves, MA (2005). Adeno-associated virus: from defective virus to effective vector. Virol J 2: 43.
6. Gao, G, Vandenberghe, LH, Alvira, MR, Lu, Y, Calcedo, R, Zhou, X et al. (2004). Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol 78: 6381-6388.
7. Wu, Z, Asokan, A and Samulski, RJ (2006). Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 14: 316-327.
8. Wu, Z, Miller, E, Agbandje-McKenna, M and Samulski, RJ (2006). Alpha2,3 and alpha2,6 N-linked sialic acids facilitate effcient binding and transduction by adeno-associated virus types 1 and 6. J Virol 80: 9093-9103.
9. Akache, B, Grimm, D, Pandey, K, Yant, SR, Xu, H and Kay, MA (2006). The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J Virol 80: 9831-9836.
10. Fisher, SK, Lewis, GP, Linberg, KA and Verardo, MR (2005). Cellular remodeling in mammalian retina: results from studies of experimental retinal detachment. Prog Retin Eye Res 24: 395-431.
11. Jacobson, SG, Aleman, TS, Cideciyan, AV, Roman, AJ, Sumaroka, A, Windsor, EA et al. (2009). Defning the residual vision in leber congenital amaurosis caused by RPE65 mutations. Invest Ophthalmol Vis Sci 50: 2368-2375.
12. Jacobson, SG, Aleman, TS, Sumaroka, A, Cideciyan, AV, Roman, AJ, Windsor, EA et al. (2009). Disease boundaries in the retina of patients with Usher syndrome caused by MYO7A gene mutations. Invest Ophthalmol Vis Sci 50: 1886-1894.
13. Jacobson, SG, Aleman, TS, Cideciyan, AV, Sumaroka, A, Schwartz, SB, Windsor, EA et al. (2005). Identifying photoreceptors in blind eyes caused by RPE65 mutations: prerequisite for human gene therapy success. Proc Natl Acad Sci USA 102: 6177-6182.
14. Maguire, AM, Simonelli, F, Pierce, EA, Pugh, EN Jr., Mingozzi, F, Bennicelli, J et al. (2008). Safety and effcacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358: 2240-2248.
15. Harvey, AR, Kamphuis, W, Eggers, R, Symons, NA, Blits, B, Niclou, S et al. (2002). Intravitreal injection of adeno-associated viral vectors results in the transduction of different types of retinal neurons in neonatal and adult rats: a comparison with lentiviral vectors. Mol Cell Neurosci 21: 141-157.
16. Candiello, J, Balasubramani, M, Schreiber, EM, Cole, GJ, Mayer, U, Halfter, W et al. (2007). Biomechanical properties of native basement membranes. FEBS J 274: 2897-2908.
17. Halfter, W, Dong, S, Dong, A, Eller, AW and Nischt, R (2008). Origin and turnover of ECM proteins from the inner limiting membrane and vitreous body. Eye 22: 1207-1213.
18. Halfter, W (1998). Disruption of the retinal basal lamina during early embryonic development leads to a retraction of vitreal end feet, an increased number of ganglion cells, and aberrant axonal outgrowth. J Comp Neurol 397: 89-104.
19. Semina, EV, Bosenko, DV, Zinkevich, NC, Soules, KA, Hyde, DR, Vihtelic, TS et al. (2006). Mutations in laminin alpha 1 result in complex, lens-independent ocular phenotypes in zebrafsh. Dev Biol 299: 63-77.
20. Mester, V and Kuhn, F (2000). Internal limiting membrane removal in the management of full-thickness macular holes. Am J Ophthalmol 129: 769-777.
21. Heegaard, S, Jensen, OA and Prause, JU (1986). Structure and composition of the inner limiting membrane of the retina. SEM on frozen resin-cracked and enzyme-digested retinas of Macaca mulatta. Graefes Arch Clin Exp Ophthalmol 224: 355-360.
22. Bartlett, JS, Wilcher, R and Samulski, RJ (2000). Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors. J Virol 74: 2777-2785.
23. Jurásek, J, Johnson, P, Olafson, RW and Smillie, LB (1971). An improved fractionation system for pronase on CM-sephadex. Can J Biochem 49: 1195-1201.
24. Van Vliet, K, Blouin, V, Agbandje-McKenna, M and Snyder, RO (2006). Proteolytic mapping of the adeno-associated virus capsid. Mol Ther 14: 809-821.
25. Surace, EM and Auricchio, A (2008). Versatility of AAV vectors for retinal gene transfer. Vision Res 48: 353-359.
26. McCarty, DM (2008). Self-complementary AAV vectors; advances and applications. Mol Ther 16: 1648-1656.
27. Schultz, BR and Chamberlain, JS (2008). Recombinant adeno-associated virus transduction and integration. Mol Ther 16: 1189-1199.
28. Allocca, M, Mussolino, C, Garcia-Hoyos, M, Sanges, D, Iodice, C, Petrillo, M et al. (2007). Novel adeno-associated virus serotypes effciently transduce murine photoreceptors. J Virol 81: 11372-11380.
29. Auricchio, A, Kobinger, G, Anand, V, Hildinger, M, O'Connor, E, Maguire, AM et al. (2001). Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 10: 3075-3081.
30. Weber, M, Rabinowitz, J, Provost, N, Conrath, H, Folliot, S, Briot, D et al. (2003). Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol Ther 7: 774-781.
31. Pickard, GE, Smeraski, CA, Tomlinson, CC, Banfeld, BW, Kaufman, J, Wilcox, CL et al. (2002). Intravitreal injection of the attenuated pseudorabies virus PRV Bartha results in infection of the hamster suprachiasmatic nucleus only by retrograde transsynaptic transport via autonomic circuits. J Neurosci 22: 2701-2710.
32. Chai, L and Morris, JE (1999). Heparan sulfate in the inner limiting membrane of embryonic chicken retina binds basic fbroblast growth factor to promote axonal outgrowth. Exp Neurol 160: 175-185.
33. Chai, L and Morris, JE (1994). Distribution of heparan sulfate proteoglycans in embryonic chicken neural retina and isolated inner limiting membrane. Curr Eye Res 13: 669-677.
34. Zhong, L, Li, B, Mah, CS, Govindasamy, L, Agbandje-McKenna, M, Cooper, M et al. (2008). Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-effciency transduction at lower doses. Proc Natl Acad Sci USA 105: 7827-7832.
35. Petrs-Silva, H, Dinculescu, A, Li, Q, Min, SH, Chiodo, V, Pang, JJ et al. (2009). High-effciency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 17: 463-471.
36. Kaludov, N, Brown, KE, Walters, RW, Zabner, J and Chiorini, JA (2001). Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and effcient transduction but differ in sialic acid linkage specificity. J Virol 75: 6884-6893.
37. Cho, EY, Choi, HL and Chan, FL (2002). Expression pattern of glycoconjugates in rat retina as analysed by lectin histochemistry. Histochem J 34: 589-600.
38. Allocca, M, Doria, M, Petrillo, M, Colella, P, Garcia-Hoyos, M, Gibbs, D et al. (2008). Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 118: 1955-1964.
39. Grieger, JC, Choi, VW and Samulski, RJ (2006). Production and characterization of adeno-associated viral vectors. Nat Protoc 1: 1412-1428.