FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2002914110> ?p ?o ?g. }

• W2002914110 endingPage "650" @default.
• W2002914110 startingPage "643" @default.
• W2002914110 abstract "The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated viral (AAV) vectors encoding RPE65 in patients affected with LCA2 due to mutations in the RPE65 gene, is safe and, in some cases, results in efficacy. We evaluated the long-term safety and efficacy (global effects on retinal/visual function) resulting from subretinal administration of AAV2-hRPE65v2. Both the safety and the efficacy noted at early timepoints persist through at least 1.5 years after injection in the three LCA2 patients enrolled in the low dose cohort of our trial. A transient rise in neutralizing antibodies to AAV capsid was observed but there was no humoral response to RPE65 protein. The persistence of functional amelioration suggests that AAV-mediated gene transfer to the human retina does not elicit immunological responses which cause significant loss of transduced cells. The persistence of physiologic effect supports the possibility that gene therapy may influence LCA2 disease progression. The safety of the intervention and the stability of the improvement in visual and retinal function in these subjects support the use of AAV-mediated gene augmentation therapy for treatment of inherited retinal diseases. The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated viral (AAV) vectors encoding RPE65 in patients affected with LCA2 due to mutations in the RPE65 gene, is safe and, in some cases, results in efficacy. We evaluated the long-term safety and efficacy (global effects on retinal/visual function) resulting from subretinal administration of AAV2-hRPE65v2. Both the safety and the efficacy noted at early timepoints persist through at least 1.5 years after injection in the three LCA2 patients enrolled in the low dose cohort of our trial. A transient rise in neutralizing antibodies to AAV capsid was observed but there was no humoral response to RPE65 protein. The persistence of functional amelioration suggests that AAV-mediated gene transfer to the human retina does not elicit immunological responses which cause significant loss of transduced cells. The persistence of physiologic effect supports the possibility that gene therapy may influence LCA2 disease progression. The safety of the intervention and the stability of the improvement in visual and retinal function in these subjects support the use of AAV-mediated gene augmentation therapy for treatment of inherited retinal diseases." @default.
• W2002914110 created "2016-06-24" @default.
• W2002914110 creator A5001681530 @default.
• W2002914110 creator A5003669388 @default.
• W2002914110 creator A5007568701 @default.
• W2002914110 creator A5017458885 @default.
• W2002914110 creator A5018276675 @default.
• W2002914110 creator A5019070349 @default.
• W2002914110 creator A5020648798 @default.
• W2002914110 creator A5036346252 @default.
• W2002914110 creator A5037182958 @default.
• W2002914110 creator A5048366850 @default.
• W2002914110 creator A5049298836 @default.
• W2002914110 creator A5049773865 @default.
• W2002914110 creator A5051555445 @default.
• W2002914110 creator A5058485837 @default.
• W2002914110 creator A5058764792 @default.
• W2002914110 creator A5060779925 @default.
• W2002914110 creator A5075244777 @default.
• W2002914110 creator A5075311667 @default.
• W2002914110 creator A5075836236 @default.
• W2002914110 creator A5084935546 @default.
• W2002914110 creator A5084980482 @default.
• W2002914110 creator A5088818305 @default.
• W2002914110 date "2010-03-01" @default.
• W2002914110 modified "2023-10-11" @default.
• W2002914110 title "Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration" @default.
• W2002914110 cites W1976329054 @default.
• W2002914110 cites W1977847843 @default.
• W2002914110 cites W1985844587 @default.
• W2002914110 cites W1995140705 @default.
• W2002914110 cites W1995915881 @default.
• W2002914110 cites W1996875707 @default.
• W2002914110 cites W1999109674 @default.
• W2002914110 cites W2001603084 @default.
• W2002914110 cites W2004906535 @default.
• W2002914110 cites W2022040013 @default.
• W2002914110 cites W2024156701 @default.
• W2002914110 cites W2035101755 @default.
• W2002914110 cites W2056320636 @default.
• W2002914110 cites W2065451039 @default.
• W2002914110 cites W2073848291 @default.
• W2002914110 cites W2128033750 @default.
• W2002914110 cites W2137222303 @default.
• W2002914110 cites W2141354154 @default.
• W2002914110 cites W2141575938 @default.
• W2002914110 cites W2141838218 @default.
• W2002914110 cites W2151809142 @default.
• W2002914110 cites W2157117004 @default.
• W2002914110 cites W2164088504 @default.
• W2002914110 cites W2164468733 @default.
• W2002914110 doi "https://doi.org/10.1038/mt.2009.277" @default.
• W2002914110 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/2839440" @default.
• W2002914110 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/19953081" @default.
• W2002914110 hasPublicationYear "2010" @default.
• W2002914110 type Work @default.
• W2002914110 sameAs 2002914110 @default.
• W2002914110 citedByCount "496" @default.
• W2002914110 countsByYear W20029141102012 @default.
• W2002914110 countsByYear W20029141102013 @default.
• W2002914110 countsByYear W20029141102014 @default.
• W2002914110 countsByYear W20029141102015 @default.
• W2002914110 countsByYear W20029141102016 @default.
• W2002914110 countsByYear W20029141102017 @default.
• W2002914110 countsByYear W20029141102018 @default.
• W2002914110 countsByYear W20029141102019 @default.
• W2002914110 countsByYear W20029141102020 @default.
• W2002914110 countsByYear W20029141102021 @default.
• W2002914110 countsByYear W20029141102022 @default.
• W2002914110 countsByYear W20029141102023 @default.
• W2002914110 crossrefType "journal-article" @default.
• W2002914110 hasAuthorship W2002914110A5001681530 @default.
• W2002914110 hasAuthorship W2002914110A5003669388 @default.
• W2002914110 hasAuthorship W2002914110A5007568701 @default.
• W2002914110 hasAuthorship W2002914110A5017458885 @default.
• W2002914110 hasAuthorship W2002914110A5018276675 @default.
• W2002914110 hasAuthorship W2002914110A5019070349 @default.
• W2002914110 hasAuthorship W2002914110A5020648798 @default.
• W2002914110 hasAuthorship W2002914110A5036346252 @default.
• W2002914110 hasAuthorship W2002914110A5037182958 @default.
• W2002914110 hasAuthorship W2002914110A5048366850 @default.
• W2002914110 hasAuthorship W2002914110A5049298836 @default.
• W2002914110 hasAuthorship W2002914110A5049773865 @default.
• W2002914110 hasAuthorship W2002914110A5051555445 @default.
• W2002914110 hasAuthorship W2002914110A5058485837 @default.
• W2002914110 hasAuthorship W2002914110A5058764792 @default.
• W2002914110 hasAuthorship W2002914110A5060779925 @default.
• W2002914110 hasAuthorship W2002914110A5075244777 @default.
• W2002914110 hasAuthorship W2002914110A5075311667 @default.
• W2002914110 hasAuthorship W2002914110A5075836236 @default.
• W2002914110 hasAuthorship W2002914110A5084935546 @default.
• W2002914110 hasAuthorship W2002914110A5084980482 @default.
• W2002914110 hasAuthorship W2002914110A5088818305 @default.
• W2002914110 hasBestOaLocation W20029141101 @default.
• W2002914110 hasConcept C104317684 @default.
• W2002914110 hasConcept C111599444 @default.
• W2002914110 hasConcept C118487528 @default.
• W2002914110 hasConcept C126322002 @default.

• next