FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W1981508914> ?p ?o ?g. }

• W1981508914 endingPage "410" @default.
• W1981508914 startingPage "404" @default.
• W1981508914 abstract "Ornithine transcarbamylase deficiency (OTCD) is the most common inborn error of urea synthesis. Complete OTCD can result in hyperammonemic coma in the neonatal period, which can rapidly become fatal. Current acute therapy involves dialysis; chronic therapy involves the stimulation of alternate nitrogen clearance pathways; and the only curative approach is liver transplantation. Adeno-associated virus (AAV) vector-based gene therapy would add to current treatment options provided the vector delivers high level and stable transgene expression in liver without dose-limiting toxicity. In this study, we employed an AAV2/8-based self-complementary (sc) vector expressing the murine OTC (mOTC) gene under a liver-specific thyroxine-binding globulin promoter and examined the therapeutic effects in a mouse model of OTCD, the spf (ash) mouse. Seven days after a single intravenous injection of vector, treated mice showed complete normalization of urinary orotic acid, a measure of OTC activity. We further improved vector efficacy by incorporating a Kozak or Kozak-like sequence into mOTC complementary DNA, which increased the OTC activity by five or twofold and achieved sustained correction of orotic aciduria for up to 7 months. Our results demonstrate that vector optimizations can significantly improve the efficacy of gene therapy." @default.
• W1981508914 created "2016-06-24" @default.
• W1981508914 creator A5009988205 @default.
• W1981508914 creator A5017675556 @default.
• W1981508914 creator A5020162592 @default.
• W1981508914 creator A5036549445 @default.
• W1981508914 creator A5046895078 @default.
• W1981508914 creator A5048362726 @default.
• W1981508914 creator A5051157484 @default.
• W1981508914 creator A5051344823 @default.
• W1981508914 creator A5074400978 @default.
• W1981508914 creator A5083739731 @default.
• W1981508914 date "2011-08-18" @default.
• W1981508914 modified "2023-09-26" @default.
• W1981508914 title "Sustained correction of OTC deficiency in spfash mice using optimized self-complementary AAV2/8 vectors" @default.
• W1981508914 cites W1543387521 @default.
• W1981508914 cites W1966994747 @default.
• W1981508914 cites W1967312147 @default.
• W1981508914 cites W1980491559 @default.
• W1981508914 cites W1988691937 @default.
• W1981508914 cites W1994495694 @default.
• W1981508914 cites W2004590552 @default.
• W1981508914 cites W2009968900 @default.
• W1981508914 cites W2017444975 @default.
• W1981508914 cites W2019868085 @default.
• W1981508914 cites W2032435016 @default.
• W1981508914 cites W2047488470 @default.
• W1981508914 cites W2047608515 @default.
• W1981508914 cites W2075638876 @default.
• W1981508914 cites W2080848255 @default.
• W1981508914 cites W2081200794 @default.
• W1981508914 cites W2090864647 @default.
• W1981508914 cites W2091134968 @default.
• W1981508914 cites W2093424805 @default.
• W1981508914 cites W2120271934 @default.
• W1981508914 cites W2121397816 @default.
• W1981508914 cites W2142334980 @default.
• W1981508914 cites W2145365087 @default.
• W1981508914 cites W2148848239 @default.
• W1981508914 cites W2151902657 @default.
• W1981508914 cites W2313914167 @default.
• W1981508914 cites W2394894015 @default.
• W1981508914 doi "https://doi.org/10.1038/gt.2011.111" @default.
• W1981508914 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3321078" @default.
• W1981508914 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/21850052" @default.
• W1981508914 hasPublicationYear "2011" @default.
• W1981508914 type Work @default.
• W1981508914 sameAs 1981508914 @default.
• W1981508914 citedByCount "32" @default.
• W1981508914 countsByYear W19815089142012 @default.
• W1981508914 countsByYear W19815089142013 @default.
• W1981508914 countsByYear W19815089142015 @default.
• W1981508914 countsByYear W19815089142016 @default.
• W1981508914 countsByYear W19815089142017 @default.
• W1981508914 countsByYear W19815089142018 @default.
• W1981508914 countsByYear W19815089142019 @default.
• W1981508914 countsByYear W19815089142020 @default.
• W1981508914 countsByYear W19815089142021 @default.
• W1981508914 countsByYear W19815089142022 @default.
• W1981508914 countsByYear W19815089142023 @default.
• W1981508914 crossrefType "journal-article" @default.
• W1981508914 hasAuthorship W1981508914A5009988205 @default.
• W1981508914 hasAuthorship W1981508914A5017675556 @default.
• W1981508914 hasAuthorship W1981508914A5020162592 @default.
• W1981508914 hasAuthorship W1981508914A5036549445 @default.
• W1981508914 hasAuthorship W1981508914A5046895078 @default.
• W1981508914 hasAuthorship W1981508914A5048362726 @default.
• W1981508914 hasAuthorship W1981508914A5051157484 @default.
• W1981508914 hasAuthorship W1981508914A5051344823 @default.
• W1981508914 hasAuthorship W1981508914A5074400978 @default.
• W1981508914 hasAuthorship W1981508914A5083739731 @default.
• W1981508914 hasBestOaLocation W19815089142 @default.
• W1981508914 hasConcept C102230213 @default.
• W1981508914 hasConcept C104317684 @default.
• W1981508914 hasConcept C111599444 @default.
• W1981508914 hasConcept C126322002 @default.
• W1981508914 hasConcept C134018914 @default.
• W1981508914 hasConcept C2775851010 @default.
• W1981508914 hasConcept C2777468819 @default.
• W1981508914 hasConcept C2778107364 @default.
• W1981508914 hasConcept C2780469793 @default.
• W1981508914 hasConcept C2911091166 @default.
• W1981508914 hasConcept C32470452 @default.
• W1981508914 hasConcept C40767141 @default.
• W1981508914 hasConcept C515207424 @default.
• W1981508914 hasConcept C55493867 @default.
• W1981508914 hasConcept C71924100 @default.
• W1981508914 hasConcept C77061444 @default.
• W1981508914 hasConcept C86803240 @default.
• W1981508914 hasConcept C92087593 @default.
• W1981508914 hasConcept C98274493 @default.
• W1981508914 hasConceptScore W1981508914C102230213 @default.
• W1981508914 hasConceptScore W1981508914C104317684 @default.
• W1981508914 hasConceptScore W1981508914C111599444 @default.
• W1981508914 hasConceptScore W1981508914C126322002 @default.
• W1981508914 hasConceptScore W1981508914C134018914 @default.
• W1981508914 hasConceptScore W1981508914C2775851010 @default.
• W1981508914 hasConceptScore W1981508914C2777468819 @default.

• next