This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Rent or buy this article
Prices vary by article type
from$1.95
to$39.95
Prices may be subject to local taxes which are calculated during checkout
References
Rodriguez-Estevez L, Asokan P, Borrás T, Transduction optimization of AAV vectors for human gene therapy of glaucoma and their reversed cell entry characteristics. Gene Ther. 2019. https://doi.org/10.1038/s41434-019-0105-4.
Borrás T, Buie LK, Spiga MG. Inducible scAAV2.GRE.MMP1 lowers IOP long-term in a large animal model for steroid-induced glaucoma gene therapy. Gene Ther. 2016;23:438–49. https://doi.org/10.1038/gt.2016.14.
Song L, Llanga T, Conatser LM, Zaric V, Gilger BC, Hirsch ML. Serotype survey of AAV gene delivery via subconjunctival injection in mice. Gene Ther. 2018;25:402–14. https://doi.org/10.1038/s41434-018-0035-6.
Nascimento-Dos-Santos G, Teixeira-Pinheiro LC, da Silva-Júnior AJ, Carvalho LRP, Mesentier-Louro LA, Hauswirth WW, et al. Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury. Gene Ther. 2019. https://doi.org/10.1038/s41434-019-0089-0.
Cao X, Yung J, Mak H, Leung CKS. Factors governing the transduction efficiency of adeno-associated virus in the retinal ganglion cells following intravitreal injection. Gene Ther. 2019;26:109–20. https://doi.org/10.1038/s41434-019-0060-0.
Dias MS, Araujo VG, Vasconcelos T, Li Q, Hauswirth WW, Linden R, et al. Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat. Gene Ther. 2019;26:479–90. https://doi.org/10.1038/s41434-019-0100-9.
Zeng Y, Qian H, Wu Z, Marangoni D, Sieving PA, Bush RA. AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration. Gene Ther. 2019;26:386–98. https://doi.org/10.1038/s41434-019-0094-3.
Sullivan JA, Stanek LM, Lukason MJ, Bu J, Osmond SR, Barry EA, et al. Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain. Gene Ther. 2018;25:205–19. https://doi.org/10.1038/s41434-018-0017-8.
Boyd RF, Boye SL, Conlon TJ, Erger KE, Sledge DG, Langohr IM, et al. Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs. Gene Ther. 2016;23:548–56. https://doi.org/10.1038/gt.2016.31.
Georgiadis A, Duran Y, Ribeiro J, Abelleira-Hervas L, Robbie SJ, Sünkel-Laing B, et al. Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65. Gene Ther. 2016;23:857–62. https://doi.org/10.1038/gt.2016.66.
Kampik D, Basche M, Luhmann UFO, Nishiguchi KM, Williams JAE, Greenwood J, et al. In situ regeneration of retinal pigment epithelium by gene transfer of E2F2: a potential strategy for treatment of macular degenerations. Gene Ther. 2017;24:810–8. https://doi.org/10.1038/gt.2017.89.
Hickey DG, Edwards TL, Barnard AR, Singh MS, de Silva SR, McClements ME, et al. Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina. Gene Ther. 2017;24:787–800. https://doi.org/10.1038/gt.2017.85.
De Silva SR, Charbel Issa P, Singh MS, Lipinski DM, Barnea-Cramer AO, Walker NJ, et al. Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex vivo. Gene Ther. 2016;23:767–74. https://doi.org/10.1038/gt.2016.54.
Lu Q, Ganjawala TH, Ivanova E, Cheng JG, Troilo D, Pan ZH. AAV-mediated transduction and targeting of retinal bipolar cells with improved mGluR6 promoters in rodents and primates. Gene Ther. 2016;23:680–9. https://doi.org/10.1038/gt.2016.42.
Sugano E, Tabata K, Takahashi M, Nishiyama F, Shimizu H, Sato M, et al. Local and systemic responses following intravitreous injection of AAV2-encoded modified Volvox channelrhodopsin-1 in a genetically blind rat model. Gene Ther. 2016;23:158–66. https://doi.org/10.1038/gt.2015.99.
Ameline B, Tshilenge KT, Weber M, Biget M, Libeau L, Caplette R, et al. Long-term expression of melanopsin and channelrhodopsin causes no gross alterations in the dystrophic dog retina. Gene Ther. 2017;24:735–41. https://doi.org/10.1038/gt.2017.63.
Funding
This study was supported by NIHR Oxford Biomedical Research Centre.
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Conflict of interest
The author is a named inventor on several retinal gene therapy patents owned by the University of Oxford. He is or has recently been a consultant in relation to retinal gene therapy to Novartis, Biogen, Spark Therapeutics, Gyroscope Therapeutics and the National Institute for Health and Care Excellence, which advises the UK National Health Service.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Rights and permissions
About this article
Cite this article
MacLaren, R.E. A 2020 vision of ocular gene therapy. Gene Ther 28, 217–219 (2021). https://doi.org/10.1038/s41434-020-0170-8
Received:
Revised:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41434-020-0170-8