A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa
نویسندگان
چکیده
منابع مشابه
A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa
Mutational heterogeneity in genes causative of dominantly inherited disorders represents a significant barrier for development of therapies directed towards correction of the primary genetic defect. To circumvent the mutational heterogeneity present in rhodopsin- (RHO-) linked autosomal dominant Retinitis Pigmentosa (adRP), a strategy involving suppression and replacement of RHO has been adopte...
متن کاملRhodopsin transgenic pigs as a model for human retinitis pigmentosa.
PURPOSE To further characterize the retinas of Pro3471Leu rhodopsin transgenic pigs, a model for human retinitis pigmentosa. METHODS Retinas from normal and transgenic pigs, newborn to 20 months old, were processed for light and electron microscopic immunocytochemical examination. RESULTS At birth, rod numbers were normal in the transgenic retinas, but their outer segments were short and di...
متن کاملGene Therapy for X-Linked Retinitis Pigmentosa
Copyright: © 2013 Shu X. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited. Retinitis Pigmentosa (RP) is a group of heterogeneous genetic disorders with a worldwide prevalence of 1 in 4000 individuals [1]. RP can...
متن کاملA retinitis pigmentosa GTPase regulator (RPGR)-deficient mouse model for X-linked retinitis pigmentosa (RP3).
The X-linked RP3 locus codes for retinitis pigmentosa GTPase regulator (RPGR), a protein of unknown function with sequence homology to the guanine nucleotide exchange factor for Ran GTPase. We created an RPGR-deficient murine model by gene knockout. In the mutant mice, cone photoreceptors exhibit ectopic localization of cone opsins in the cell body and synapses and rod photoreceptors have a red...
متن کاملGene Therapy for Retinitis Pigmentosa
The retina comprises diverse differentiated neurons that have specific functions. Photoreceptor cells, the first-order neurons in the retina, have photopigments (rhodopsin and opsin) that absorb photons. Signals produced by the photoreceptor cells are transmitted to second-order neurons. Finally, visual signals are transmitted to the brain from the third-order neurons, the retinal ganglion cell...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Vision Research
سال: 2008
ISSN: 0042-6989
DOI: 10.1016/j.visres.2007.08.014