AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

PLoS ONE

Volumn 8, Issue 1, 2013, Pages

  Vandenberghe, Luk H ^a,c   Bell, Peter ^a   Maguire, Albert M ^b   Xiao, Ru ^a,c   Hopkins, Tim B ^b,d   Grant, Rebecca ^a   Bennett, Jean ^b   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d Uniformed Services University of the Health Sciences and the Walter Reed National Military Medical Center   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRAL VECTOR 7; ADENO ASSOCIATED VIRAL VECTOR 8; ADENO ASSOCIATED VIRAL VECTOR 9; CYTOMEGALOVIRUS VECTOR; ENHANCED GREEN FLUORESCENT PROTEIN; GALACTOSYLATED GLYCAN; GLYCAN DERIVATIVE; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL EXPERIMENT; ARTICLE; CELL BASED GENE THERAPY; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; FEMALE; GENE DOSAGE; GENE EXPRESSION; MACACA; MALE; NONHUMAN; QUANTITATIVE ANALYSIS; RETINA; RETINA CONE; RETINA FOVEA; RETINA PARAFOVEA; RETINA PERIFOVEA; RETINA ROD; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM; VIRUS CAPSID;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; LEBER CONGENITAL AMAUROSIS; PRIMATES; RETINA; RETINAL CONE PHOTORECEPTOR CELLS; RETINAL ROD PHOTORECEPTOR CELLS; TRANSDUCTION, GENETIC;

EID: 84873830812     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0053463     Document Type: Article

References (29)

1. Milam AH, Li ZY, Fariss RN, (1998) Histopathology of the human retina in retinitis pigmentosa. Prog Retin Eye Res 17: 175-205.
2. Hamel CP, (2007) Cone rod dystrophies. Orphanet J Rare Dis 2: 7.
3. Pang JJ, Alexander J, Lei B, Deng W, Zhang K, et al. (2010) Achromatopsia as a potential candidate for gene therapy. Adv Exp Med Biol. 664: 639-646.
4. Jacobson SG, Cideciyan AV, (2010) Treatment possibilities for retinitis pigmentosa. N Engl J Med 363: 1669-1671.
5. Busskamp V, Duebel J, Balya D, Fradot M, Viney TJ, et al. (2010) Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science 329: 413-417.
6. Vandenberghe LH, Bell P, Maguire AM, Cearley CN, Xiao R, et al. (2011) Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey. Sci Transl Med 3(88): 88ra54.
7. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, et al. (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358: 2231-2239.
8. Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, 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.
9. Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, et al. (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358: 2240-2248.
10. den Hollander AI, Black A, Bennett J, Cremers FP, (2010) Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies. J Clin Invest 120: 3042-3053.
11. Bennett J, Maguire AM, Cideciyan AV, Schnell M, Glover E, et al. (1999) Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci U S A 96: 9920-9925.
12. Bainbridge JW, Mistry A, Schlichtenbrede FC, Smith A, Broderick C, et al. (2003) Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina. Gene Ther 10: 1336-1344.
13. Beltran WA, Boye SL, Boye SE, Chiodo VA, Lewin AS, et al. (2010) rAAV2/5 gene-targeting to rods: dose-dependent efficiency and complications associated with different promoters. Gene Ther 17: 1162-1174.
14. Boye SE, Alexander JJ, Boye SL, Witherspoon CD, Sandefer K, et al. (2012) The human rhodopsin kinase promoter in an AAV5 vector confers rod and cone specific expression in the primate retina. Hum Gene Ther 23: 1101-1115.
15. Komaromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, et al. (2010) Gene therapy rescues cone function in congenital achromatopsia. Hum Mol Genet 19: 2581-2593.
16. Mancuso K, Hauswirth WW, Li Q, Connor TB, Kuchenbecker JA, et al. (2009) Gene therapy for red-green colour blindness in adult primates. Nature 461: 784-787.
17. Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, et al. (2007) Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol 81: 11372-11380.
18. Lebherz C, Maguire A, Tang W, Bennett J, Wilson JM, (2008) Novel AAV serotypes for improved ocular gene transfer. J Gene Med 10: 375-382.
19. Stieger K, Colle MA, Dubreil L, Mendes-Madeira A, Weber M, et al. (2008) Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol Ther 16: 916-923.
20. Mussolino C, della Corte M, Rossi S, Viola F, Di Vicino U, et al. (2011) AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Ther 18: 637-645.
21. Bell CL, Vandenberghe LH, Bell P, Limberis MP, Gao GP, et al. (2011) The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J Clin Invest 121: 2427-2435.
22. Summerford C, Samulski RJ, (1998) Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 72: 1438-1445.
23. Kaludov N, Brown KE, Walters RW, Zabner J, Chiorini JA, (2001) Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity. J Virol 75: 6884-6893.
24. Wu Z, Miller E, Agbandje-McKenna M, Samulski RJ, (2006) Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6. J Virol 80: 9093-9103.
25. Vandenberghe LH, Breous E, Nam HJ, Gao G, Xiao R, et al. (2009) Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints. Gene Ther 16: 1416-1428.
26. Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, et al. (2004) Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol 78: 6381-6388.
27. Shen S, Bryant KD, Brown SM, Randell SH, Asokan A, (2011) Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. J Biol Chem 286: 13532-13540.
28. Blanks JC, Johnson LV, (1984) Specific binding of peanut lectin to a class of retinal photoreceptor cells. A species comparison. Invest Ophthalmol Vis Sci 25: 546-557.
29. Vandenberghe LH, Xiao R, Lock M, Lin J, Korn M, et al. (2010) Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. Hum Gene Ther 21: 1251-1257.