FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2100093768> ?p ?o ?g. }

• W2100093768 endingPage "578" @default.
• W2100093768 startingPage "571" @default.
• W2100093768 abstract "Pompe disease is a lysosomal storage disease caused by the absence of acid alpha-1,4 glucosidase (GAA). The pathophysiology of Pompe disease includes generalized myopathy of both cardiac and skeletal muscle. We sought to use recombinant adeno-associated virus (rAAV) vectors to deliver functional GAA genes in vitro and in vivo. Myotubes and fibroblasts from Pompe patients were transduced in vitro with rAAV2-GAA. At 14 days postinfection, GAA activities were at least fourfold higher than in their respective untransduced controls, with a 10-fold increase observed in GAA-deficient myotubes. BALB/c and Gaa(-/-) mice were also treated with rAAV vectors. Persistent expression of vector-derived human GAA was observed in BALB/c mice up to 6 months after treatment. In Gaa(-/-) mice, intramuscular and intramyocardial delivery of rAAV2-Gaa (carrying the mouse Gaa cDNA) resulted in near-normal enzyme activities. Skeletal muscle contractility was partially restored in the soleus muscles of treated Gaa(-/-) mice, indicating the potential for vector-mediated restoration of both enzymatic activity and muscle function. Furthermore, intramuscular treatment with a recombinant AAV serotype 1 vector (rAAV1-Gaa) led to nearly eight times normal enzymatic activity in Gaa(-/-) mice, with concomitant glycogen clearance as assessed in vitro and by proton magnetic resonance spectroscopy." @default.
• W2100093768 created "2016-06-24" @default.
• W2100093768 creator A5001108635 @default.
• W2100093768 creator A5024753271 @default.
• W2100093768 creator A5029214396 @default.
• W2100093768 creator A5035164479 @default.
• W2100093768 creator A5048651139 @default.
• W2100093768 creator A5049408185 @default.
• W2100093768 creator A5056507445 @default.
• W2100093768 creator A5058934103 @default.
• W2100093768 creator A5061131591 @default.
• W2100093768 creator A5066693493 @default.
• W2100093768 creator A5072518895 @default.
• W2100093768 creator A5077382358 @default.
• W2100093768 date "2002-05-01" @default.
• W2100093768 modified "2023-10-01" @default.
• W2100093768 title "Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors" @default.
• W2100093768 cites W1508384479 @default.
• W2100093768 cites W1519383882 @default.
• W2100093768 cites W169655665 @default.
• W2100093768 cites W1786920819 @default.
• W2100093768 cites W1842480026 @default.
• W2100093768 cites W1963623233 @default.
• W2100093768 cites W1967357094 @default.
• W2100093768 cites W1971873142 @default.
• W2100093768 cites W1974877896 @default.
• W2100093768 cites W1975117307 @default.
• W2100093768 cites W1978519692 @default.
• W2100093768 cites W1979746830 @default.
• W2100093768 cites W1988631157 @default.
• W2100093768 cites W1988798016 @default.
• W2100093768 cites W1990177188 @default.
• W2100093768 cites W1997538716 @default.
• W2100093768 cites W2007854533 @default.
• W2100093768 cites W2008887811 @default.
• W2100093768 cites W2014010760 @default.
• W2100093768 cites W2019247095 @default.
• W2100093768 cites W2020814705 @default.
• W2100093768 cites W2020970293 @default.
• W2100093768 cites W2023796460 @default.
• W2100093768 cites W2028607742 @default.
• W2100093768 cites W2030487155 @default.
• W2100093768 cites W2030827836 @default.
• W2100093768 cites W2035499535 @default.
• W2100093768 cites W2050245606 @default.
• W2100093768 cites W2051159468 @default.
• W2100093768 cites W2058350049 @default.
• W2100093768 cites W2059108053 @default.
• W2100093768 cites W2062006811 @default.
• W2100093768 cites W2065704959 @default.
• W2100093768 cites W2081487415 @default.
• W2100093768 cites W2095093948 @default.
• W2100093768 cites W2106911160 @default.
• W2100093768 cites W2109305605 @default.
• W2100093768 cites W2127608615 @default.
• W2100093768 cites W2139493402 @default.
• W2100093768 cites W2146999721 @default.
• W2100093768 cites W2148223197 @default.
• W2100093768 cites W2154736819 @default.
• W2100093768 cites W2155142318 @default.
• W2100093768 cites W2163435252 @default.
• W2100093768 cites W2199171497 @default.
• W2100093768 cites W2432911140 @default.
• W2100093768 cites W39175234 @default.
• W2100093768 cites W4211260918 @default.
• W2100093768 doi "https://doi.org/10.1006/mthe.2002.0580" @default.
• W2100093768 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/11991748" @default.
• W2100093768 hasPublicationYear "2002" @default.
• W2100093768 type Work @default.
• W2100093768 sameAs 2100093768 @default.
• W2100093768 citedByCount "120" @default.
• W2100093768 countsByYear W21000937682012 @default.
• W2100093768 countsByYear W21000937682013 @default.
• W2100093768 countsByYear W21000937682014 @default.
• W2100093768 countsByYear W21000937682015 @default.
• W2100093768 countsByYear W21000937682016 @default.
• W2100093768 countsByYear W21000937682017 @default.
• W2100093768 countsByYear W21000937682018 @default.
• W2100093768 countsByYear W21000937682019 @default.
• W2100093768 countsByYear W21000937682020 @default.
• W2100093768 countsByYear W21000937682021 @default.
• W2100093768 countsByYear W21000937682022 @default.
• W2100093768 countsByYear W21000937682023 @default.
• W2100093768 crossrefType "journal-article" @default.
• W2100093768 hasAuthorship W2100093768A5001108635 @default.
• W2100093768 hasAuthorship W2100093768A5024753271 @default.
• W2100093768 hasAuthorship W2100093768A5029214396 @default.
• W2100093768 hasAuthorship W2100093768A5035164479 @default.
• W2100093768 hasAuthorship W2100093768A5048651139 @default.
• W2100093768 hasAuthorship W2100093768A5049408185 @default.
• W2100093768 hasAuthorship W2100093768A5056507445 @default.
• W2100093768 hasAuthorship W2100093768A5058934103 @default.
• W2100093768 hasAuthorship W2100093768A5061131591 @default.
• W2100093768 hasAuthorship W2100093768A5066693493 @default.
• W2100093768 hasAuthorship W2100093768A5072518895 @default.
• W2100093768 hasAuthorship W2100093768A5077382358 @default.
• W2100093768 hasBestOaLocation W21000937681 @default.
• W2100093768 hasConcept C104317684 @default.

• next