Wong KA, Benowitz LI. Retinal ganglion cell survival and axon regeneration after optic nerve injury: role of inflammation and other factors. Int J Mol Sci. 2022;23:10179.
Article CAS PubMed PubMed Central Google Scholar
Sanz-Morello B, Ahmadi H, Vohra R, Saruhanian S, Freude KK, Hamann S, et al. Oxidative stress in optic neuropathies. Antioxidants. 2021;10:1538.
Article CAS PubMed PubMed Central Google Scholar
Newman NJ, Yu-Wai-Man P, Biousse V, Carelli V. Understanding the molecular basis and pathogenesis of hereditary optic neuropathies: towards improved diagnosis and management. Lancet Neurol. 2023;22:172–88.
Article CAS PubMed Google Scholar
Quigley HA. Glaucoma. Lancet. 2011;377:1367–77.
Shu DY, Chaudhary S, Cho KS, Lennikov A, Miller WP, Thorn DC, et al. Role of oxidative stress in ocular diseases: a balancing act. Metabolites. 2023;13:187.
Article CAS PubMed PubMed Central Google Scholar
Fague L, Liu YA, Marsh-Armstrong N. The basic science of optic nerve regeneration. Ann Transl Med. 2021;9:1276.
Article CAS PubMed PubMed Central Google Scholar
Levin LA, Patrick C, Choudry NB, Sharif NA, Goldberg JL. Neuroprotection in neurodegenerations of the brain and eye: lessons from the past and directions for the future. Front Neurol. 2022;13:964197.
Article PubMed PubMed Central Google Scholar
Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358–78.
Article CAS PubMed PubMed Central Google Scholar
Zinn E, Vandenberghe LH. Adeno-associated virus: fit to serve. Curr Opin Virol. 2014;8:90–7.
Cwerman-Thibault H, Augustin S, Ellouze S, Sahel JA, Corral-Debrinski M. Gene therapy for mitochondrial diseases: leber hereditary optic neuropathy as the first candidate for a clinical trial. C R Biol. 2014;337:193–206.
Martin KR, Quigley HA. Gene therapy for optic nerve disease. Eye. 2004;18:1049–55.
Article CAS PubMed Google Scholar
Mak KY, Rajapaksha IG, Angus PW, Herath CB. The adeno-associated virus - a safe and promising vehicle for liverspecific gene therapy of inherited and non-inherited disorders. Curr Gene Ther. 2017;17:4–16.
Article CAS PubMed Google Scholar
Bennett J. Taking stock of retinal gene therapy: looking back and moving forward. Mol Ther. 2017;25:1076–94.
Article CAS PubMed PubMed Central Google Scholar
Gao J, Hussain RM, Weng CY. Voretigene neparvovec in retinal diseases: a review of the current clinical evidence. Clin Ophthalmol. 2020;14:3855–69.
Article CAS PubMed PubMed Central Google Scholar
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390:849–60.
Article CAS PubMed PubMed Central Google Scholar
Buning H, Perabo L, Coutelle O, Quadt-Humme S, Hallek M. Recent developments in adeno-associated virus vector technology. J Gene Med. 2008;10:717–33.
Srivastava A. In vivo tissue-tropism of adeno-associated viral vectors. Curr Opin Virol. 2016;21:75–80.
Article CAS PubMed PubMed Central Google Scholar
O’Donnell J, Taylor KA, Chapman MS. Adeno-associated virus-2 and its primary cellular receptor–Cryo-EM structure of a heparin complex. Virology. 2009;385:434–43.
Hadaczek P, Mirek H, Bringas J, Cunningham J, Bankiewicz K. Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain. Hum Gene Ther. 2004;15:469–79.
Article CAS PubMed Google Scholar
Kashiwakura Y, Tamayose K, Iwabuchi K, Hirai Y, Shimada T, Matsumoto K, et al. Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection. J Virol. 2005;79:609–14.
Article CAS PubMed PubMed Central Google Scholar
Asokan A, Hamra JB, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry. J Virol. 2006;80:8961–9.
Article CAS PubMed PubMed Central Google Scholar
Kurzeder C, Koppold B, Sauer G, Pabst S, Kreienberg R, Deissler H. CD9 promotes adeno-associated virus type 2 infection of mammary carcinoma cells with low cell surface expression of heparan sulphate proteoglycans. Int J Mol Med. 2007;19:325–33.
Van Vliet KM, Blouin V, Brument N, Agbandje-McKenna M, Snyder RO. The role of the adeno-associated virus capsid in gene transfer. Methods Mol Biol. 2008;437:51–91.
Article PubMed PubMed Central Google Scholar
Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X, 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. 2002;76:791–801.
Article CAS PubMed PubMed Central Google Scholar
Ramachandran PS, Lee V, Wei Z, Song JY, Casal G, Cronin T, et al. Evaluation of dose and safety of AAV7m8 and AAV8BP2 in the non-human primate retina. Hum Gene Ther. 2017;28:154–67.
Article CAS PubMed PubMed Central Google Scholar
Ross AG, Chaqour B, McDougald DS, Dine KE, Duong TT, Shindler RE, et al. Selective upregulation of sirt1 expression in retinal ganglion cells by AAV-mediated gene delivery increases neuronal cell survival and alleviates axon demyelination associated with optic neuritis. Biomolecules. 2022;12:830.
Article CAS PubMed PubMed Central Google Scholar
Yue J, Khan RS, Duong TT, Dine KE, Cui QN, O’Neill N, et al. Cell-specific expression of human sirt1 by gene therapy reduces retinal ganglion cell loss induced by elevated intraocular pressure. Neurotherapeutics. 2023;20:896–907.
Article CAS PubMed Google Scholar
McDougald DS, Dine KE, Zezulin AU, Bennett J, Shindler KS. SIRT1 and NRF2 gene transfer mediate distinct neuroprotective effects upon retinal ganglion cell survival and function in experimental optic neuritis. Invest Ophthalmol Vis Sci. 2018;59:1212–20.
Article CAS PubMed PubMed Central Google Scholar
Alam F, Syed H, Amjad S, Baig M, Khan TA, Rehman R. Interplay between oxidative stress, SIRT1, reproductive and metabolic functions. Curr Res Physiol. 2021;4:119–24.
Article CAS PubMed PubMed Central Google Scholar
Singh V, Ubaid S. Role of silent information regulator 1 (SIRT1) in regulating oxidative stress and inflammation. Inflammation. 2020;43:1589–98.
Article CAS PubMed Google Scholar
Li L, Zhi D, Cheng R, Li J, Luo C, Li H. The neuroprotective role of SIRT1/PGC-1alpha signaling in limb postconditioning in cerebral ischemia/reperfusion injury. Neurosci Lett. 2021;749:135736.
Article CAS PubMed Google Scholar
Nita M, Grzybowski A. Interplay between reactive oxygen species and autophagy in the course of age-related macular degeneration. EXCLI J. 2020;19:1353–71.
PubMed PubMed Central Google Scholar
Samarin J, Wessel J, Cicha I, Kroening S, Warnecke C, Goppelt-Struebe M. FoxO proteins mediate hypoxic induction of connective tissue growth factor in endothelial cells. J Biol Chem. 2010;285:4328–36.
Article CAS PubMed Google Scholar
Chaffiol A, Caplette R, Jaillard C, Brazhnikova E, Desrosiers M, Dubus E, et al. A new promoter allows optogenetic vision restoration with enhanced sensitivity in macaque retina. Mol Ther. 2017;25:2546–60.
Article CAS PubMed PubMed Central Google Scholar
Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, et al. Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther. 2008;16:458–65.
留言 (0)