Advances in AAV vector development for gene therapy in the retina

Advances in Experimental Medicine and Biology

Volumn 801, Issue , 2014, Pages 687-693

  Day, Timothy P ^a   Byrne, Leah C ^a   Schaffer, David V ^a   Flannery, John G ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adeno associated virus; Directed evolution; Gene therapy; Mutagenesis; Retinal degeneration

Indexed keywords

DNA; PARVOVIRUS VECTOR; VASCULOTROPIN; VASCULOTROPIN RECEPTOR 1;

AGE RELATED MACULAR DEGENERATION; ARTICLE; CHOROIDEREMIA; DRUG EFFICACY; DRUG SAFETY; FLT1 GENE; GENE; GENE EXPRESSION; GENETIC ENGINEERING; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HUMAN; NONHUMAN; OUTCOME ASSESSMENT; PIGMENT EPITHELIUM; PRIORITY JOURNAL; RETINA DEGENERATION; TRANSGENE; VEGF GENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM; VIRUS CAPSID; ADENO ASSOCIATED VIRUS; ANIMAL; GENE THERAPY; GENE VECTOR; GENETICS; METHODOLOGY; PHYSIOLOGY; RETINA; RETINA DISEASE; REVIEW;

ADENO-ASSOCIATED VIRUS; ANIMALIA; DEPENDOVIRUS;

ANIMALS; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; RETINA; RETINAL DISEASES;

EID: 84904817762     PISSN: 00652598     EISSN: 22148019     Source Type: Book Series    
DOI: 10.1007/978-1-4614-3209-8_86     Document Type: Article

References (31)

1. Aslanidi GV, Rivers AE, Ortiz L, Govindasamy L, Ling C, Jayandharan GR et al (2012) High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine 30(26):3908-3917. doi:10.1016/j.vaccine.2012.03.079
2. Bainbridge JWB, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K et al (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358(21):2231-2239. doi:10.1056/NEJMoa0802268
3. Bartel MA, Weinstein JR, Schaffer DV (2012) Directed evolution of novel adeno-associated viruses for therapeutic gene delivery. Gene Ther 19(6):694-700. doi:10.1038/gt.2012.20
4. Cepko CL (2012) Emerging gene therapies for retinal degenerations. J Neurosci 32(19):6415-6420. doi:10.1523/JNEUROSCI.0295-12.2012
5. Dalkara D, Byrne LC, Klimczak RR, Visel M, Yu L, Merigan WH, Flannery JG, Schaffer DV (2013) In vivo directed evolution of a novel adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med 5(189): 189ra76 (Manuscript submitted to Science translational medicine)
6. 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(9):3042-3053. doi:10.1172/JCI42258
7. Fields BN, Knipe DM, Howley PM (2007) Fields virology, 5th edn. Lippincott Williams & Wilkins, Philadelphia
8. Goncalves MA (2005) Adeno-associated virus: from defective virus to effective vector. Virol J 2:43
9. Grimm D, Kay MA (2003) From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 3(4):281-304. http://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom =pubmed&id=12871018&retmode=ref&cmd=prlinks
10. 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(10):979-990. doi:10.1089/hum.2008.107
11. Klimczak RR, Koerber JT, Dalkara D, Flannery JG, Schaffer DV (2009) A novel adenoassociated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PLoS ONE 4(10):e7467. doi:10.1371/journal.pone.0007467
12. Koerber JT, Schaffer DV (2008) Transposon-based mutagenesis generates diverse adenoassociated viral libraries with novel gene delivery properties. Methods Mol Biol. Totowa, NJ: Humana Press, pp 161-170. doi:10.1007/978-1-60327-248-3_10
13. Koerber JT, Jang J-H, Schaffer DV (2008) DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol Ther 16(10):1703-1709. doi:10.1038/mt.2008.167
14. Koilkonda RD, Chou T-H, Porciatti V, Hauswirth WW, Guy J (2010) Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus. Arch Ophthalmol 128(7):876-883. doi:10.1001/archophthalmol.2010.135
15. Ku CA, Chiodo VA, Boye SL, Goldberg AFX, Li TT, Hauswirth WW, Ramamurthy V (2011) Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Hum Mol Genet 20(23):4569-4581. doi:10.1093/hmg/ddr391
16. Lukason M, DuFresne E, Rubin H, Pechan P, Li Q, Kim I et al (2011) Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule. Mol Ther 19(2):260-265. doi:10.1038/ mt.2010.230
17. MacLachlan TK, Lukason M, Collins M, Munger R, Isenberger E, Rogers C et al (2011) Preclinical Safety Evaluation of AAV2-sFLT01-A gene therapy for age-related macular degeneration. Mol Ther 19(2):326-334. doi:10.1038/mt.2010.258
18. Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J et al (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358(21):2240-2248. doi:10.1056/NEJMoa0802315
19. Maheshri N, Koerber JT, Kaspar BK, Schaffer DV (2006) Directed evolution of adenoassociated virus yields enhanced gene delivery vectors. Nat Biotechnol 24(2):198-204. doi:10.1038/nbt1182
20. McCarty DM, Monahan PE, Samulski RJ (2001) Self-complementary recombinant adenoassociated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Ther 8(16):1248-1254. doi:10.1038/sj.gt.3301514
21. Natkunarajah M, Trittibach P, McIntosh J, DURAN Y, Barker SE, Smith AJ et al (2007) Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Ther 15(6):463-467. doi:10.1038/sj.gt.3303074
22. Pang J, Boye SE, Lei B, Boye SL, Everhart D, Ryals R et al (2010). Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Ther 17(7):815-826. doi:10.1038/gt.2010.29
23. Perabo L, Endell J, King S, Lux K, Goldnau D, Hallek M, Büning H (2006) Combinatorial engineering of a gene therapy vector: directed evolution of adeno-associated virus. J Gene Med 8(2):155-162. doi:10.1002/jgm.849
24. Petersen-Jones SM, Bartoe JT, Fischer AJ, Scott M, Boye SL, CHIODO V, Hauswirth WW (2009) AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector. Mol Vision 15:1835-1842
25. Petrs-Silva H, Dinculescu A, Li Q, Min S-H, Chiodo V, Pang JJ et al (2008) High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 17(3):463-471. doi:10.1038/mt.2008.269
26. Simonelli FF, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL et al (2010) Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 18(3):643-650. doi:10.1038/mt.2009.277
27. Sonntag F, Schmidt K, Kleinschmidt JA. (2010) A viral assembly factor promotes AAV2 capsid formation in the nucleolus. Proc Natl Acad of Sci U S A 107(22):10220-10225. doi:10.1073/pnas.1001673107
28. Vandenberghe LH, Auricchio A (2011) Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther 19(2):162-168. doi:10.1038/gt.2011.151
29. Vandenberghe LH, Wilson JM, Gao G (2009) Tailoring the AAV vector capsid for gene therapy. Gene Ther 16(3):311-319. doi:10.1038/gt.2008.170
30. Wu J, Zhao W, Zhong L, Han Z, Li B, Ma W et al (2007) Self-complementary recombinant adeno-associated viral vectors: packaging capacity and the role of rep proteins in vector purity. Hum Gene Ther 18(2):171-182. doi:10.1089/hum.2006.088
31. Zhong LL, Li BB, Mah CSC, Govindasamy LL, Agbandje-McKenna MM, Cooper MM et al (2008) Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A 105(22):7827-7832. doi:10.1073/pnas.0802866105