FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2030065638> ?p ?o ?g. }

• W2030065638 endingPage "133" @default.
• W2030065638 startingPage "127" @default.
• W2030065638 abstract "A biodistribution and toxicology study was performed to test the acute toxicities of intradiaphragmatic injection of a recombinant adeno-associated virus (rAAV) 2/1-human acid alpha-Glucosidase (hGAA) driven by a cytomegalovirus (CMV) promoter (rAAV1-CMV-hGAA) in New Zealand white rabbits and in the rodent Pompe disease model by injecting at the right quadriceps. Studies performed using fluoroscopy and AAV2-GFP demonstrated spread upon intradiaphragmatic injection, and the ability of AAV to infect and express acid α-glucosidase (GAA) throughout the diaphragm. For the preclinical study, 10 rabbits (5 male, 5 female) were divided into two groups, vehicle control (Lactated Ringer's) and test article (1.5×1012 vector genomes [vg] rAAV1-CMV-hGAA), and euthanized on day 21. After direct visualization, the left hemidiaphragm was injected at three locations. There was up to a 2,500-fold increase in circulating anti-AAV1 antibodies directed to the vector capsids. In addition, up to an 18-fold increase in antibodies against the GAA protein was generated. Injection sites maintained up to 1.0×105 vg/μg genomic DNA (gDNA), while uninjected sites had up to 1.0×104 vg/μg gDNA. Vector DNA was present in blood at 24 hr postinjection at up to 1.0×106 vg/μg gDNA, followed by a decrease to 1.0×103 vg/μg gDNA at euthanization on day 21. Nominal amounts of vector DNA were present in peripheral organs, including the brain, spinal cord, gonads, and skeletal muscle. Upon histopathological examination, fibroplasias of the serosal surface were noted at diaphragm injections sites of both groups. In addition, an increase in mononuclear cell infiltration in the diaphragm and esophagus in vector-dosed animals was found. Elevated creatine phosphokinase levels, an indicator of muscle repair, was observed in all animals postprocedure but persisted in vector-injected rabbits until euthanization. A follow-up study suggested that this was directed against the human transgene expression in a foreign species. Overall, this study demonstrates diffusion of vector throughout the diaphragm after localized injections. Conlon and colleagues report results from a biodistribution and toxicology study designed to test the acute toxicities of an rAAV2/1-hGAA vector driven by a cytomegalovirus promoter (rAAV1-CMVhGAA) in New Zealand White rabbits and in the rodent Pompe disease model. Their studies demonstrate that localized injection in rabbits leads to diffusion of vector throughout the diaphragm and that intramuscular injection leads to a biodistribution profile similar to that observed in other AAV1 intramuscular preclinical studies." @default.
• W2030065638 created "2016-06-24" @default.
• W2030065638 creator A5001210643 @default.
• W2030065638 creator A5014115936 @default.
• W2030065638 creator A5014684785 @default.
• W2030065638 creator A5015775536 @default.
• W2030065638 creator A5015923505 @default.
• W2030065638 creator A5022886286 @default.
• W2030065638 creator A5029214396 @default.
• W2030065638 creator A5047851542 @default.
• W2030065638 creator A5052819930 @default.
• W2030065638 creator A5062727443 @default.
• W2030065638 creator A5064422415 @default.
• W2030065638 creator A5069186905 @default.
• W2030065638 date "2013-09-01" @default.
• W2030065638 modified "2023-10-14" @default.
• W2030065638 title "Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-Glucosidase" @default.
• W2030065638 cites W1964843260 @default.
• W2030065638 cites W1973065533 @default.
• W2030065638 cites W1973977374 @default.
• W2030065638 cites W1986979860 @default.
• W2030065638 cites W1993627451 @default.
• W2030065638 cites W2004099834 @default.
• W2030065638 cites W2008033906 @default.
• W2030065638 cites W2008895870 @default.
• W2030065638 cites W2049504823 @default.
• W2030065638 cites W2050774634 @default.
• W2030065638 cites W2055655054 @default.
• W2030065638 cites W2056320636 @default.
• W2030065638 cites W2081487415 @default.
• W2030065638 cites W2083867423 @default.
• W2030065638 cites W2084276666 @default.
• W2030065638 cites W2091777991 @default.
• W2030065638 cites W2094062778 @default.
• W2030065638 cites W2100093768 @default.
• W2030065638 cites W2115510312 @default.
• W2030065638 cites W2127608615 @default.
• W2030065638 cites W2134392644 @default.
• W2030065638 cites W2150843111 @default.
• W2030065638 cites W3105570083 @default.
• W2030065638 cites W48439084 @default.
• W2030065638 cites W80513504 @default.
• W2030065638 doi "https://doi.org/10.1089/humc.2013.147" @default.
• W2030065638 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/4003472" @default.
• W2030065638 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/24021025" @default.
• W2030065638 hasPublicationYear "2013" @default.
• W2030065638 type Work @default.
• W2030065638 sameAs 2030065638 @default.
• W2030065638 citedByCount "17" @default.
• W2030065638 countsByYear W20300656382014 @default.
• W2030065638 countsByYear W20300656382015 @default.
• W2030065638 countsByYear W20300656382016 @default.
• W2030065638 countsByYear W20300656382017 @default.
• W2030065638 countsByYear W20300656382018 @default.
• W2030065638 countsByYear W20300656382019 @default.
• W2030065638 countsByYear W20300656382021 @default.
• W2030065638 countsByYear W20300656382022 @default.
• W2030065638 crossrefType "journal-article" @default.
• W2030065638 hasAuthorship W2030065638A5001210643 @default.
• W2030065638 hasAuthorship W2030065638A5014115936 @default.
• W2030065638 hasAuthorship W2030065638A5014684785 @default.
• W2030065638 hasAuthorship W2030065638A5015775536 @default.
• W2030065638 hasAuthorship W2030065638A5015923505 @default.
• W2030065638 hasAuthorship W2030065638A5022886286 @default.
• W2030065638 hasAuthorship W2030065638A5029214396 @default.
• W2030065638 hasAuthorship W2030065638A5047851542 @default.
• W2030065638 hasAuthorship W2030065638A5052819930 @default.
• W2030065638 hasAuthorship W2030065638A5062727443 @default.
• W2030065638 hasAuthorship W2030065638A5064422415 @default.
• W2030065638 hasAuthorship W2030065638A5069186905 @default.
• W2030065638 hasBestOaLocation W20300656382 @default.
• W2030065638 hasConcept C104317684 @default.
• W2030065638 hasConcept C111599444 @default.
• W2030065638 hasConcept C153911025 @default.
• W2030065638 hasConcept C159047783 @default.
• W2030065638 hasConcept C17757408 @default.
• W2030065638 hasConcept C185592680 @default.
• W2030065638 hasConcept C202751555 @default.
• W2030065638 hasConcept C202878990 @default.
• W2030065638 hasConcept C2522874641 @default.
• W2030065638 hasConcept C2777807558 @default.
• W2030065638 hasConcept C40767141 @default.
• W2030065638 hasConcept C552990157 @default.
• W2030065638 hasConcept C55493867 @default.
• W2030065638 hasConcept C71924100 @default.
• W2030065638 hasConcept C86803240 @default.
• W2030065638 hasConcept C92087593 @default.
• W2030065638 hasConcept C98274493 @default.
• W2030065638 hasConceptScore W2030065638C104317684 @default.
• W2030065638 hasConceptScore W2030065638C111599444 @default.
• W2030065638 hasConceptScore W2030065638C153911025 @default.
• W2030065638 hasConceptScore W2030065638C159047783 @default.
• W2030065638 hasConceptScore W2030065638C17757408 @default.
• W2030065638 hasConceptScore W2030065638C185592680 @default.
• W2030065638 hasConceptScore W2030065638C202751555 @default.
• W2030065638 hasConceptScore W2030065638C202878990 @default.
• W2030065638 hasConceptScore W2030065638C2522874641 @default.
• W2030065638 hasConceptScore W2030065638C2777807558 @default.

• next