Gene therapy for the treatment of X-linked retinitis pigmentosa
نویسندگان
چکیده
منابع مشابه
Gene 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 کاملX-linked retinitis pigmentosa.
Of 107 consecutive patients with genetically-determined retinitis pigmentosa, 23 were provisionally diagnosed as having inherited the disease in an X-linked fashion. 42 affected males and 61 females were examined, and from the data obtained the following conclusions were drawn: (1) X-linked retinitis pigmentosa exists and is distinct from choroideremia. (2) In contrast to the results of previou...
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 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 کاملA novel locus for X-linked retinitis pigmentosa.
INTRODUCTION Retinitis pigmentosa (RP) is the most prevalent group of inherited retinopathies and demonstrates considerable clinical and genetic heterogeneity, with wide variations in disease severity, progression, and gene involvement. We studied a large family with RP to determine the pattern of inheritance and to identify the disease-causing gene/locus. MATERIALS AND METHODS Ophthalmic exa...
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ژورنال
عنوان ژورنال: Expert Opinion on Orphan Drugs
سال: 2018
ISSN: 2167-8707
DOI: 10.1080/21678707.2018.1444476