FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2574334611> ?p ?o ?g. }

• W2574334611 endingPage "223" @default.
• W2574334611 startingPage "215" @default.
• W2574334611 abstract "CLN2 neuronal ceroid lipofuscinosis is a hereditary lysosomal storage disease with primarily neurological signs that results from mutations in TPP1, which encodes the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Studies using a canine model for this disorder demonstrated that delivery of TPP1 enzyme to the cerebrospinal fluid (CSF) by intracerebroventricular administration of an AAV-TPP1 vector resulted in substantial delays in the onset and progression of neurological signs and prolongation of life span. We hypothesized that the treatment may not deliver therapeutic levels of this protein to tissues outside the central nervous system that also require TPP1 for normal lysosomal function. To test this hypothesis, dogs treated with CSF administration of AAV-TPP1 were evaluated for the development of non-neuronal pathology. Affected treated dogs exhibited progressive cardiac pathology reflected by elevated plasma cardiac troponin-1, impaired cardiac function and development of histopathological myocardial lesions. Progressive increases in the plasma activity levels of alanine aminotransferase and creatine kinase indicated development of pathology in the liver and muscles. The treatment also did not prevent disease-related accumulation of lysosomal storage bodies in the heart or liver. These studies indicate that optimal treatment outcomes for CLN2 disease may require delivery of TPP1 systemically as well as directly to the central nervous system." @default.
• W2574334611 created "2017-01-26" @default.
• W2574334611 creator A5002088032 @default.
• W2574334611 creator A5007663874 @default.
• W2574334611 creator A5031083398 @default.
• W2574334611 creator A5044788722 @default.
• W2574334611 creator A5057837906 @default.
• W2574334611 creator A5071434182 @default.
• W2574334611 creator A5074790517 @default.
• W2574334611 creator A5077459265 @default.
• W2574334611 date "2017-02-02" @default.
• W2574334611 modified "2023-10-02" @default.
• W2574334611 title "Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression" @default.
• W2574334611 cites W1602997642 @default.
• W2574334611 cites W1607986775 @default.
• W2574334611 cites W1625648367 @default.
• W2574334611 cites W1840644602 @default.
• W2574334611 cites W1963866355 @default.
• W2574334611 cites W1969849606 @default.
• W2574334611 cites W1973576032 @default.
• W2574334611 cites W1976010578 @default.
• W2574334611 cites W1976498837 @default.
• W2574334611 cites W1980286081 @default.
• W2574334611 cites W1985349046 @default.
• W2574334611 cites W1995642612 @default.
• W2574334611 cites W1996254410 @default.
• W2574334611 cites W2004311445 @default.
• W2574334611 cites W2006353731 @default.
• W2574334611 cites W2007320580 @default.
• W2574334611 cites W2011145479 @default.
• W2574334611 cites W2011950472 @default.
• W2574334611 cites W2014128251 @default.
• W2574334611 cites W2014894652 @default.
• W2574334611 cites W2025684242 @default.
• W2574334611 cites W2026420637 @default.
• W2574334611 cites W2032265763 @default.
• W2574334611 cites W2036844023 @default.
• W2574334611 cites W2041582307 @default.
• W2574334611 cites W2048909195 @default.
• W2574334611 cites W2051018924 @default.
• W2574334611 cites W2052298217 @default.
• W2574334611 cites W2052820865 @default.
• W2574334611 cites W2052979981 @default.
• W2574334611 cites W2062018052 @default.
• W2574334611 cites W2075110986 @default.
• W2574334611 cites W2079877218 @default.
• W2574334611 cites W2088587774 @default.
• W2574334611 cites W2109078396 @default.
• W2574334611 cites W2118655979 @default.
• W2574334611 cites W2149328369 @default.
• W2574334611 cites W2161109927 @default.
• W2574334611 cites W2163490348 @default.
• W2574334611 cites W2174260594 @default.
• W2574334611 cites W2330469059 @default.
• W2574334611 cites W2382534732 @default.
• W2574334611 cites W2396808893 @default.
• W2574334611 cites W2412411450 @default.
• W2574334611 cites W2422703843 @default.
• W2574334611 cites W4234399520 @default.
• W2574334611 cites W4235656202 @default.
• W2574334611 cites W4236630084 @default.
• W2574334611 cites W4297923741 @default.
• W2574334611 cites W430977535 @default.
• W2574334611 doi "https://doi.org/10.1038/gt.2017.4" @default.
• W2574334611 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5398942" @default.
• W2574334611 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/28079862" @default.
• W2574334611 hasPublicationYear "2017" @default.
• W2574334611 type Work @default.
• W2574334611 sameAs 2574334611 @default.
• W2574334611 citedByCount "33" @default.
• W2574334611 countsByYear W25743346112017 @default.
• W2574334611 countsByYear W25743346112018 @default.
• W2574334611 countsByYear W25743346112019 @default.
• W2574334611 countsByYear W25743346112020 @default.
• W2574334611 countsByYear W25743346112021 @default.
• W2574334611 countsByYear W25743346112022 @default.
• W2574334611 countsByYear W25743346112023 @default.
• W2574334611 crossrefType "journal-article" @default.
• W2574334611 hasAuthorship W2574334611A5002088032 @default.
• W2574334611 hasAuthorship W2574334611A5007663874 @default.
• W2574334611 hasAuthorship W2574334611A5031083398 @default.
• W2574334611 hasAuthorship W2574334611A5044788722 @default.
• W2574334611 hasAuthorship W2574334611A5057837906 @default.
• W2574334611 hasAuthorship W2574334611A5071434182 @default.
• W2574334611 hasAuthorship W2574334611A5074790517 @default.
• W2574334611 hasAuthorship W2574334611A5077459265 @default.
• W2574334611 hasBestOaLocation W25743346111 @default.
• W2574334611 hasConcept C126322002 @default.
• W2574334611 hasConcept C134018914 @default.
• W2574334611 hasConcept C142724271 @default.
• W2574334611 hasConcept C2777404818 @default.
• W2574334611 hasConcept C2778230176 @default.
• W2574334611 hasConcept C2778264360 @default.
• W2574334611 hasConcept C2778650512 @default.
• W2574334611 hasConcept C2779134260 @default.
• W2574334611 hasConcept C2779969927 @default.
• W2574334611 hasConcept C36880943 @default.
• W2574334611 hasConcept C529278444 @default.

• next