Effect of gene therapy on visual function in Leber's congenital amaurosis

JWB Bainbridge, AJ Smith, SS Barker… - … England Journal of …, 2008 - Mass Medical Soc
… retinal function and improved vision, as determined by visual mobility in low light. The purpose
of this study was to determine whether gene therapy … of efficacy in terms of visual function. …

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

LS Carvalho, J Xu, RA Pearson, AJ Smith… - Human molecular …, 2011 - academic.oup.com
… This study represents achievement of the most substantial restoration of visual function
reported to date in an animal model of achromatopsia using a human gene construct, which has …

The effect of human gene therapy for RPE65-associated Leber's congenital amaurosis on visual function: a systematic review and meta-analysis

X Wang, C Yu, RT Tzekov, Y Zhu, W Li - Orphanet Journal of Rare …, 2020 - Springer
… caused by the mutations of RPE65 gene and gene therapy has been developed to be a …
changes in visual function and application of gene therapy in patients with RPE65-LCA. …

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

KJ Wert, RJ Davis, J Sancho-Pelluz… - Human molecular …, 2013 - academic.oup.com
… is a progressive loss of photoreceptors and visual function, at a faster rate of degeneration
… /8 gene therapy vector can increase both photoreceptor survival and neuronal function in a …

The human visual cortex responds to gene therapy–mediated recovery of retinal function

M Ashtari, LL Cyckowski, JF Monroe… - The Journal of …, 2011 - Am Soc Clin Investig
visual function using one or more measures, including visual … Future studies will involve fMRI
before and after gene therapy in … of gene therapy for LCA2. Finally, the fMRI finding that the …

Gene therapy for visual loss: Opportunities and concerns

JH Lee, JH Wang, J Chen, F Li, TL Edwards… - Progress in retinal and …, 2019 - Elsevier
… have been developed in the area of gene therapy for ocular disease. We also discuss the
challenges and opportunities facing gene therapy for ocular diseases, as well as its prospects. …

Systemic adeno-associated virus-mediated gene therapy preserves retinal ganglion cells and visual function in DBA/2J glaucomatous mice

TA Sullivan, EE Geisert, J Hines-Beard… - Human gene therapy, 2011 - liebertpub.com
… In addition to anatomical preservation of both axons and somata, we found that gene
therapy using either Epo vector also preserved visual function in DBA/2J mice. F-VEPs were …

Correlation between photoreceptor layer integrity and visual function in patients with Stargardt disease: implications for gene therapy

F Testa, S Rossi, A Sodi, I Passerini… - … & visual science, 2012 - iovs.arvojournals.org
… to central atrophy and thus loss of central visual function. It has been recognized that STGD
is associated with mutations in the ABCA4 gene, which encodes a transport protein located …

Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis

ML Batten, Y Imanishi, DC Tu, T Doan, L Zhu… - PLoS …, 2005 - journals.plos.org
… could rescue visual function by pharmacological intervention and gene transfer therapy in
… of oral administration of retinoids and gene therapy. Comparison of these two approaches is …

Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy

SR De Silva, AR Barnard, S Hughes… - Proceedings of the …, 2017 - National Acad Sciences
… an immune response when introduced via gene therapy. Furthermore, this method of …
expression and restoration of visual function, indicating that this therapy could be stable and …