FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2059601221> ?p ?o ?g. }

• W2059601221 endingPage "318" @default.
• W2059601221 startingPage "311" @default.
• W2059601221 abstract "Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into the blood for up to 11 years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to +4 (severely abnormal). At 1 year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3 ± 0.7, 2.3 ± 0.6, 1.8 ± 0.5, and 1.6 ± 0.7, respectively, which were higher than the values of 0.6 ± 0.1, 0.1 ± 0.4, 0.3 ± 0.8, and 0.1 ± 0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores in their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at ≥ 8 years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and the aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11 years, and propose that neonatal initiation of ERT should have a similar effect." @default.
• W2059601221 created "2016-06-24" @default.
• W2059601221 creator A5005288385 @default.
• W2059601221 creator A5006061010 @default.
• W2059601221 creator A5022249737 @default.
• W2059601221 creator A5027440159 @default.
• W2059601221 creator A5047419053 @default.
• W2059601221 creator A5062390152 @default.
• W2059601221 creator A5079582260 @default.
• W2059601221 creator A5091231981 @default.
• W2059601221 date "2013-11-01" @default.
• W2059601221 modified "2023-10-09" @default.
• W2059601221 title "The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade" @default.
• W2059601221 cites W1558789523 @default.
• W2059601221 cites W1605694349 @default.
• W2059601221 cites W1943855526 @default.
• W2059601221 cites W1968642565 @default.
• W2059601221 cites W1977499972 @default.
• W2059601221 cites W1982338435 @default.
• W2059601221 cites W1990211122 @default.
• W2059601221 cites W1992628415 @default.
• W2059601221 cites W2006797531 @default.
• W2059601221 cites W2008316788 @default.
• W2059601221 cites W2009504318 @default.
• W2059601221 cites W2013018264 @default.
• W2059601221 cites W2014839633 @default.
• W2059601221 cites W2030179332 @default.
• W2059601221 cites W2033777138 @default.
• W2059601221 cites W2037275256 @default.
• W2059601221 cites W2042023036 @default.
• W2059601221 cites W2050557934 @default.
• W2059601221 cites W2052314207 @default.
• W2059601221 cites W2058217076 @default.
• W2059601221 cites W2063222638 @default.
• W2059601221 cites W2064851167 @default.
• W2059601221 cites W2073620213 @default.
• W2059601221 cites W2076633759 @default.
• W2059601221 cites W2085237653 @default.
• W2059601221 cites W2085987206 @default.
• W2059601221 cites W2093722439 @default.
• W2059601221 cites W2124000504 @default.
• W2059601221 cites W2124073245 @default.
• W2059601221 cites W2133111666 @default.
• W2059601221 cites W2141084326 @default.
• W2059601221 cites W2161767084 @default.
• W2059601221 doi "https://doi.org/10.1016/j.ymgme.2013.06.015" @default.
• W2059601221 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3800273" @default.
• W2059601221 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/23860311" @default.
• W2059601221 hasPublicationYear "2013" @default.
• W2059601221 type Work @default.
• W2059601221 sameAs 2059601221 @default.
• W2059601221 citedByCount "13" @default.
• W2059601221 countsByYear W20596012212014 @default.
• W2059601221 countsByYear W20596012212015 @default.
• W2059601221 countsByYear W20596012212017 @default.
• W2059601221 countsByYear W20596012212019 @default.
• W2059601221 countsByYear W20596012212020 @default.
• W2059601221 countsByYear W20596012212022 @default.
• W2059601221 crossrefType "journal-article" @default.
• W2059601221 hasAuthorship W2059601221A5005288385 @default.
• W2059601221 hasAuthorship W2059601221A5006061010 @default.
• W2059601221 hasAuthorship W2059601221A5022249737 @default.
• W2059601221 hasAuthorship W2059601221A5027440159 @default.
• W2059601221 hasAuthorship W2059601221A5047419053 @default.
• W2059601221 hasAuthorship W2059601221A5062390152 @default.
• W2059601221 hasAuthorship W2059601221A5079582260 @default.
• W2059601221 hasAuthorship W2059601221A5091231981 @default.
• W2059601221 hasBestOaLocation W20596012212 @default.
• W2059601221 hasConcept C104317684 @default.
• W2059601221 hasConcept C111599444 @default.
• W2059601221 hasConcept C126322002 @default.
• W2059601221 hasConcept C126348684 @default.
• W2059601221 hasConcept C164705383 @default.
• W2059601221 hasConcept C185592680 @default.
• W2059601221 hasConcept C2777543888 @default.
• W2059601221 hasConcept C2778264360 @default.
• W2059601221 hasConcept C2779134260 @default.
• W2059601221 hasConcept C2779416428 @default.
• W2059601221 hasConcept C2779559920 @default.
• W2059601221 hasConcept C2779969927 @default.
• W2059601221 hasConcept C2780168065 @default.
• W2059601221 hasConcept C2780714102 @default.
• W2059601221 hasConcept C2780966522 @default.
• W2059601221 hasConcept C2910727661 @default.
• W2059601221 hasConcept C2993373945 @default.
• W2059601221 hasConcept C55493867 @default.
• W2059601221 hasConcept C71924100 @default.
• W2059601221 hasConcept C86803240 @default.
• W2059601221 hasConceptScore W2059601221C104317684 @default.
• W2059601221 hasConceptScore W2059601221C111599444 @default.
• W2059601221 hasConceptScore W2059601221C126322002 @default.
• W2059601221 hasConceptScore W2059601221C126348684 @default.
• W2059601221 hasConceptScore W2059601221C164705383 @default.
• W2059601221 hasConceptScore W2059601221C185592680 @default.
• W2059601221 hasConceptScore W2059601221C2777543888 @default.
• W2059601221 hasConceptScore W2059601221C2778264360 @default.
• W2059601221 hasConceptScore W2059601221C2779134260 @default.
• W2059601221 hasConceptScore W2059601221C2779416428 @default.

• next