Caspase inhibition with xiap as an adjunct to aav vector gene-replacement therapy: Improving efficacy and prolonging the treatment window

PLoS ONE

Volumn 7, Issue 5, 2012, Pages

  Yao, Jingyu ^a   Jia, Lin ^a   Khan, Naheed ^a   Zheng, Qiong Duan ^a   Moncrief, Ashley ^a   Hauswirth, William W ^b   Thompson, Debra A ^a   Zacks, David N ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

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

Author keywords

[No Author keywords available]

Indexed keywords

CASPASE; CYCLIC GMP PHOSPHODIESTERASE; PARVOVIRUS VECTOR; PHOSPHODIESTERASE INHIBITOR; PROTEIN P21; RHODOPSIN; X LINKED INHIBITOR OF APOPTOSIS; PDE6B PROTEIN, MOUSE; PHOSPHODIESTERASE VI;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL COUNT; CELL PROTECTION; CONTROLLED STUDY; DRUG EFFICACY; ELECTRORETINOGRAM; ENZYME ACTIVITY; ENZYME INHIBITION; FEMALE; GENE REPLACEMENT THERAPY; HISTOLOGY; IMMUNOHISTOCHEMISTRY; MOUSE; NONHUMAN; PHOTORECEPTOR CELL; PROTEIN LOCALIZATION; RETINA DEGENERATION; TREATMENT RESPONSE; WESTERN BLOTTING; ADENO ASSOCIATED VIRUS; ANIMAL; BIOSYNTHESIS; C57BL MOUSE; DISEASE MODEL; DRUG ANTAGONISM; ELECTRORETINOGRAPHY; GENE THERAPY; GENE VECTOR; GENETICS; LIGHT; METABOLISM; METHODOLOGY;

ANIMALIA; MUS;

ANIMALS; CASPASES; CYCLIC NUCLEOTIDE PHOSPHODIESTERASES, TYPE 6; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ELECTRORETINOGRAPHY; GENE THERAPY; GENETIC VECTORS; LIGHT; MICE; MICE, INBRED C57BL; PHOTORECEPTOR CELLS, VERTEBRATE; RETINAL DEGENERATION; X-LINKED INHIBITOR OF APOPTOSIS PROTEIN;

EID: 84861210826     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0037197     Document Type: Article

References (26)

1. Pagon PA, (1988) Retinitis pigmentosa. Surv Ophthalmol 13: 137-177.
2. Marigo V, (2007) Programmed cell death in retinal degeneration. Targeting apoptosis in photoreceptors as potential therapy for retinal degeneration. Cell Cycle 6: 652-655.
3. Sieving PA, Caruso RC, Tao W, Coleman HR, Thompson DJS, et al. (2006) Ciliary neurotrophic factor (CNTF) for human retinal degeneration: Phase I trial of CNTF delivered by encapsulated cell intraocular implants. Proc Nat Acad Sci, USA 103: 3896-3901.
4. Talcott KE, Ratnam K, Sundquist SM, Lucero AS, Lujan BJ, et al. (2011) Longitudinal study of cone photoreceptors during retinal degeneration and in response to ciliary neurotrophic factor treatment. Invest Ophthalmol Vis Sci 52: 2219-2226.
5. Bainbridge JWB, Smith AJ, Barker SS, Robbie S, Henderson R, et al. (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. New Eng J Med 358: 2231-2239.
6. Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, et al. (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. New Eng J Med 258: 2240-2248.
7. Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, et al. (2012) Gene therapy for Leber congenital amaurosis caused by RPE65 mutations. Safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol 130: 9-24.
8. Daiger S, Sullivan L, Rossiter B, (2011) RetNet: Retinal Information Network. Available:http://www.sph.uth.tmc.edu/Retnet/.
9. Holcik M, Gibson H, Korneluk RG, (2011) XIAP: apoptotic brake and promising therapeutic target. Apoptosis 4: 253-261.
10. Renwick J, Narang MA, Coupland SG, Xuan JY, Baker AN, et al. (2006) XIAP-mediated neuroprotection in retinal ischemia. Gene Ther 13: 339-347.
11. Kugler S, Straten G, Kreppel F, Isenmann S, Liston P, et al. (2007) The X-linked inhibitor of apoptosis (XIAP) prevents cell death in axotomized CNS neurons in vivo. Cell Death Differ 7: 815-824.
12. Straten G, Schmeer C, Kretz A, Gerhardt E, Kugler S, et al. (2002) Potential synergistic protection of retinal ganglion cells from axotomy-induced apoptosis by adenoviral administration of glial cell line-derived neurotrophic factor and X-chromosome-linked inhibitor of apoptosis. Neurobiol Dis 11: 123-133.
13. McKinnon SJ, Lehman DM, Tahzib NG, Ransom NL, Reitsamer HA, et al. (2002) Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. Mol Ther 5: 780-787.
14. Cooper LB, Chan DK, Roediger FC, Shaffer BR, Fraser JF, et al. (2006) AAV-mediated delivery of the caspase inhibitor XIAP protects against cisplatin ototoxicity. Otol Neurotol 27: 484-490.
15. Petrin D, Baker A, Coupland SG, Liston P, Narang M, et al. (2003) Structural and functional protection of photoreceptors from MNU-induced retinal degeneration by the X-linked inhibitor of apoptosis. Invest Ophthalmol Vis Sci 44: 2757-2763.
16. Zadro-Lamoureux LA, Zacks DN, Baker AN, Zheng QD, Hauswirth WW, et al. (2009) XIAP effects on retinal detachment-induced photoreceptor apoptosis. Invest Ophthalmol Vis Sci 50: 1448-1452.
17. Yao J, Feathers KL, Khanna H, Thompson D, Tsilfidis C, et al. (2011) XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina. Invest Ophthalmol Vis Sci 52: 1567-1572.
18. Leonard KC, Petrin D, Coupland SG, Baker A, Leonard BC, et al. (2007) XIAP protection of photoreceptors in animal models of retinitis pigmentosa. PLoS ONE 2: e314.
19. Chang B, Hawes NL, Hurd RE, Davisson MT, Nusinowitz S, et al. (2002) Retinal degeneration mutants in the mouse. Vision Res 42: 517-525.
20. Gargini C, Terzibasi E, Mazzoni F, Strettoi E, (2006) Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: a morphological and ERG study. J Comp Neurol 500: 222-238.
21. Chang B, Hawes NL, Pardue MT, German AM, Hurd RE, et al. (2007) Two mouse retinal degenerations caused by missense mutations in the beta-subunit of rod cGMP phosphodiesterase gene. Vision Res 47: 624-633.
22. Pang JJ, Boye SL, Kumar A, Dinculescu A, Deng W, et al. (2008) AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEβ mutation. Invest Ophthalmol Vis Sci 49: 4278-4283.
23. Pang J, Dai X, Boye SE, Barone I, Boye SL, et al. (2011) Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa. Mol Ther 19: 234-243.
24. Muradov H, Boyd KK, Artemyev NO, (2010) Rod phosphodiesterase-6 PDE6A and PDE6B subunits are enzymatically equivalent. J Biol Chem 285: 39828-39834.
25. Sancho-Pelluz J, Arango-Gonzalez B, Kustermann S, Romero FJ, van Veen T, et al. (2008) Photoreceptor cell death mechanisms in inherited retinal degenerations. Mol Neurobiol 38: 253-269.
26. Fisher SK, Lewis GP, Linberg KA, Verardo MR, (2005) Cellular remodeling in mammalian retina: results from studies of experimental retinal detachment. Prog Retin Eye Res 24: 395-431.