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AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia
It is shown that gene therapy leads to significant rescue of cone-mediated ERGs, normal visual acuities and contrast sensitivities, and the results provide the foundation for development of an AAV5-based gene therapy trial for human CNGA3 achromatopsia. Expand
Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia.
AAV-mediated CNGA3 expression can restore cone function and rescue structure following IVit delivery of AAV8 (Y447, 733F) vector, providing a potential alternative vector delivery mode for safely transducing foveal cones in achromatopsia patients and in other human retinal diseases affecting fovea function. Expand
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.
It is suggested that tyrosine-capsid mutant AAV vectors may be effective for treating other rapidly degenerating models of retinal degeneration and the first demonstration of long-term phenotypic rescue by gene therapy in an animal model of PDEβ-RP. Expand
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.
To clarify whether transduction efficiency and cell type specificity of self-complementary (sc) AAV5 vectors are similar to those of standard, single-stranded AAV5 vectors in normal retina, one microExpand
Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia
AAV8 with capsid Y-F and T-V mutations may be one of the most effective AAV vectors for long-term treatment in a naturally occurring mouse model of CNGA3 achromatopsia. Expand
Histone Deacetylases Inhibitors in the Treatment of Retinal Degenerative Diseases: Overview and Perspectives
The neuroprotective effects of common HDACs in retinal degenerative diseases are summarized and a prospect to the applications of HDACis in the treatment of retinaldegenerative diseases in the future is made. Expand
Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye
The results show that an initial intravitreal injection of AAV vectors to one eye of a mouse does not influence AAV-mediated gene expression or related therapeutic effects in the other eye when vectors are administered to the subretinal space, and suggests that the sub retinal space possesses a unique immune privilege relative to the vitreous cavity. Expand
Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis.
The results support and extend those of the previous study that gene therapy can stop early cone degeneration, and provide proof that delayed treatment can restore the function and morphology of the remaining cones. Expand
Review: The history and role of naturally occurring mouse models with Pde6b mutations
Viral vector, especially adeno-associated viral vector (AAV) -mediated gene replacement therapy, pharmacological treatment, cell-based therapy and other approaches that extend the therapeutic window of treatment, is a potentially promising strategy for improving photoreceptor function and significantly slowing the process of retinal degeneration. Expand
Photoreceptor degeneration in a new Cacna1f mutant mouse model
A new Cacna1f mutation is discovered in nob9 mice, which display more severe phenotypes than do nob2 mice, and causes a degeneration of the outer plexiform layer in this mouse model. Expand