FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2014611118> ?p ?o ?g. }

• W2014611118 endingPage "323" @default.
• W2014611118 startingPage "311" @default.
• W2014611118 abstract "The ability of AAV2/5 to target murine photoreceptors was used to optimize vector expression cassettes for rhodopsin replacement gene therapy in the retina. Defects in this pivotal protein in the visual cycle are associated with various forms of inherited eye defects including retinitis pigmentosa. A final optimized vector design led to functional correction and regeneration of rod outer segment in a mouse model of rhodopsin deficiency. The rhodopsin gene (RHO) encodes a highly expressed G protein-coupled receptor that is central to visual transduction in rod photoreceptors. A suite of recombinant 2/5 adeno-associated viral (AAV) RHO replacement vectors has been generated in an attempt to recapitulate endogenous rhodopsin levels from exogenously delivered AAV vectors in the retina of mice with a targeted disruption in the rhodopsin gene (Rho–/– mice). Approximately 40% of wild-type mouse rhodopsin mRNA levels (RNA taken from whole retinas) was achieved in vivo in AAV-RHO-injected eyes, representing approximately 50-fold increases in expression compared with the initial vector. The main focus of this study was to test whether expression of AAV-RHO replacement in Rho–/– mice provided therapeutic benefit, which to date had not been achieved. Rho–/– mice neither elaborate rod outer segments nor have rod-derived electroretinograms (ERGs). Our results indicate for the first time in this model that subretinal AAV-RHO delivery leads not only to RHO immunolabeling but the generation of rod outer segments as evaluated by light and transmission electron microscopy. Improved histology was accompanied by rod photoreceptor activity as assessed by ERG for at least 12 weeks postinjection. The most efficient AAV-RHO constructs presented in this study provide sufficient levels of RHO to be of therapeutic benefit in Rho–/– mice and therefore represent important steps toward generating potent AAV-RHO replacement genes for gene therapy in RHO-linked human retinopathies." @default.
• W2014611118 created "2016-06-24" @default.
• W2014611118 creator A5024289997 @default.
• W2014611118 creator A5026138139 @default.
• W2014611118 creator A5033371311 @default.
• W2014611118 creator A5036824809 @default.
• W2014611118 creator A5039510197 @default.
• W2014611118 creator A5044868212 @default.
• W2014611118 creator A5055627888 @default.
• W2014611118 creator A5055993566 @default.
• W2014611118 creator A5076908022 @default.
• W2014611118 creator A5080599699 @default.
• W2014611118 creator A5084222497 @default.
• W2014611118 date "2010-03-01" @default.
• W2014611118 modified "2023-10-03" @default.
• W2014611118 title "Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene" @default.
• W2014611118 cites W1544899066 @default.
• W2014611118 cites W1845093339 @default.
• W2014611118 cites W1966368993 @default.
• W2014611118 cites W1966617699 @default.
• W2014611118 cites W1968800607 @default.
• W2014611118 cites W1970286448 @default.
• W2014611118 cites W1975554944 @default.
• W2014611118 cites W1982062477 @default.
• W2014611118 cites W1996064503 @default.
• W2014611118 cites W2004331116 @default.
• W2014611118 cites W2004635090 @default.
• W2014611118 cites W2015176049 @default.
• W2014611118 cites W2017471396 @default.
• W2014611118 cites W2022040013 @default.
• W2014611118 cites W2023349789 @default.
• W2014611118 cites W2029862966 @default.
• W2014611118 cites W2033248675 @default.
• W2014611118 cites W2033393155 @default.
• W2014611118 cites W2035898194 @default.
• W2014611118 cites W2036012055 @default.
• W2014611118 cites W2045031393 @default.
• W2014611118 cites W2047506789 @default.
• W2014611118 cites W2049890303 @default.
• W2014611118 cites W2052658245 @default.
• W2014611118 cites W2058095641 @default.
• W2014611118 cites W2062740687 @default.
• W2014611118 cites W2066098514 @default.
• W2014611118 cites W2073658331 @default.
• W2014611118 cites W2075929828 @default.
• W2014611118 cites W2077508733 @default.
• W2014611118 cites W2079178498 @default.
• W2014611118 cites W2080775648 @default.
• W2014611118 cites W2087185853 @default.
• W2014611118 cites W2102120200 @default.
• W2014611118 cites W2113903634 @default.
• W2014611118 cites W2118200380 @default.
• W2014611118 cites W2128166968 @default.
• W2014611118 cites W2135252826 @default.
• W2014611118 cites W2137222303 @default.
• W2014611118 cites W2140128594 @default.
• W2014611118 cites W2141354154 @default.
• W2014611118 cites W2141818804 @default.
• W2014611118 cites W2142334980 @default.
• W2014611118 cites W2143841965 @default.
• W2014611118 cites W2150788231 @default.
• W2014611118 cites W2151235295 @default.
• W2014611118 cites W2151809142 @default.
• W2014611118 cites W2160119905 @default.
• W2014611118 cites W2162478996 @default.
• W2014611118 cites W2162811183 @default.
• W2014611118 cites W2169961890 @default.
• W2014611118 doi "https://doi.org/10.1089/hum.2009.119" @default.
• W2014611118 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/19824806" @default.
• W2014611118 hasPublicationYear "2010" @default.
• W2014611118 type Work @default.
• W2014611118 sameAs 2014611118 @default.
• W2014611118 citedByCount "52" @default.
• W2014611118 countsByYear W20146111182012 @default.
• W2014611118 countsByYear W20146111182013 @default.
• W2014611118 countsByYear W20146111182014 @default.
• W2014611118 countsByYear W20146111182015 @default.
• W2014611118 countsByYear W20146111182016 @default.
• W2014611118 countsByYear W20146111182017 @default.
• W2014611118 countsByYear W20146111182018 @default.
• W2014611118 countsByYear W20146111182019 @default.
• W2014611118 countsByYear W20146111182020 @default.
• W2014611118 countsByYear W20146111182021 @default.
• W2014611118 countsByYear W20146111182022 @default.
• W2014611118 countsByYear W20146111182023 @default.
• W2014611118 crossrefType "journal-article" @default.
• W2014611118 hasAuthorship W2014611118A5024289997 @default.
• W2014611118 hasAuthorship W2014611118A5026138139 @default.
• W2014611118 hasAuthorship W2014611118A5033371311 @default.
• W2014611118 hasAuthorship W2014611118A5036824809 @default.
• W2014611118 hasAuthorship W2014611118A5039510197 @default.
• W2014611118 hasAuthorship W2014611118A5044868212 @default.
• W2014611118 hasAuthorship W2014611118A5055627888 @default.
• W2014611118 hasAuthorship W2014611118A5055993566 @default.
• W2014611118 hasAuthorship W2014611118A5076908022 @default.
• W2014611118 hasAuthorship W2014611118A5080599699 @default.
• W2014611118 hasAuthorship W2014611118A5084222497 @default.
• W2014611118 hasConcept C103796816 @default.

• next