FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2076123467> ?p ?o ?g. }

• W2076123467 endingPage "418" @default.
• W2076123467 startingPage "407" @default.
• W2076123467 abstract "Glycogen storage disease type Ia (GSD-Ia) is the inherited deficiency of glucose-6-phosphatase (G6Pase), primarily found in liver and kidney, which causes life-threatening hypoglycemia. Dogs with GSD-Ia were treated with double-stranded adeno-associated virus (AAV) vectors encoding human G6Pase. Administration of an AAV9 pseudotyped (AAV2/9) vector to seven consecutive GSD-Ia neonates prevented hypoglycemia during fasting for up to 8 hr; however, efficacy eventually waned between 2 and 30 months of age, and readministration of a new pseudotype was eventually required to maintain control of hypoglycemia. Three of these dogs succumbed to acute hypoglycemia between 7 and 9 weeks of age; however, this demise could have been prevented by earlier readministration an AAV vector, as demonstrated by successful prevention of mortality of three dogs treated earlier in life. Over the course of this study, six out of nine dogs survived after readministration of an AAV vector. Of these, each dog required readministration on average every 9 months. However, two were not retreated until >34 months of age, while one with preexisting antibodies was re-treated three times in 10 months. Glycogen content was normalized in the liver following vector administration, and G6Pase activity was increased in the liver of vector-treated dogs in comparison with GSD-Ia dogs that received only with dietary treatment. G6Pase activity reached approximately 40% of normal in two female dogs following AAV2/9 vector administration. Elevated aspartate transaminase in absence of inflammation indicated that hepatocellular turnover in the liver might drive the loss of vector genomes. Survival was prolonged for up to 60 months in dogs treated by readministration, and all dogs treated by readministration continue to thrive despite the demonstrated risk for recurrent hypoglycemia and mortality from waning efficacy of the AAV2/9 vector. These preclinical data support the further translation of AAV vector–mediated gene therapy in GSD-Ia. Demaster and colleagues report preclinical results of treating dogs with glycogen storage disease type Ia (GSD-Ia) with double-stranded adeno-associated viral vector type 9 (AAV2/9) encoding human glucose-6-phosphatase (G6Pase). Vector treatment was able to prevent hypoglycemia and led to normalized liver glycogen levels and increased G6Pase activity, but carefully timed readministration with a different pseudotype was required to prevent the therapeutic effect from waning. Survival was prolonged for up to 60 months in dogs treated by readministration, with all dogs continuing to thrive despite the demonstrated risk for recurrent hypoglycemia and mortality from waning vector efficacy." @default.
• W2076123467 created "2016-06-24" @default.
• W2076123467 creator A5004705861 @default.
• W2076123467 creator A5008425789 @default.
• W2076123467 creator A5010311721 @default.
• W2076123467 creator A5012885531 @default.
• W2076123467 creator A5015904630 @default.
• W2076123467 creator A5016713193 @default.
• W2076123467 creator A5018959877 @default.
• W2076123467 creator A5040756867 @default.
• W2076123467 creator A5045419573 @default.
• W2076123467 creator A5049814810 @default.
• W2076123467 creator A5053464984 @default.
• W2076123467 creator A5077666402 @default.
• W2076123467 creator A5077884718 @default.
• W2076123467 creator A5083923366 @default.
• W2076123467 date "2012-04-01" @default.
• W2076123467 modified "2023-10-14" @default.
• W2076123467 title "Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia" @default.
• W2076123467 cites W1943985333 @default.
• W2076123467 cites W1974168476 @default.
• W2076123467 cites W1976329054 @default.
• W2076123467 cites W1980424821 @default.
• W2076123467 cites W1983230618 @default.
• W2076123467 cites W1987013465 @default.
• W2076123467 cites W1989065400 @default.
• W2076123467 cites W2000185853 @default.
• W2076123467 cites W2022418798 @default.
• W2076123467 cites W2025246242 @default.
• W2076123467 cites W2038532989 @default.
• W2076123467 cites W2047608515 @default.
• W2076123467 cites W2052329219 @default.
• W2076123467 cites W2052905641 @default.
• W2076123467 cites W2054620742 @default.
• W2076123467 cites W2057111254 @default.
• W2076123467 cites W2070756046 @default.
• W2076123467 cites W2079806155 @default.
• W2076123467 cites W2081637766 @default.
• W2076123467 cites W2087916154 @default.
• W2076123467 cites W2104913285 @default.
• W2076123467 cites W2149562376 @default.
• W2076123467 cites W2151514148 @default.
• W2076123467 cites W2158967767 @default.
• W2076123467 cites W2166330663 @default.
• W2076123467 doi "https://doi.org/10.1089/hum.2011.106" @default.
• W2076123467 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/4047999" @default.
• W2076123467 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/22185325" @default.
• W2076123467 hasPublicationYear "2012" @default.
• W2076123467 type Work @default.
• W2076123467 sameAs 2076123467 @default.
• W2076123467 citedByCount "28" @default.
• W2076123467 countsByYear W20761234672012 @default.
• W2076123467 countsByYear W20761234672013 @default.
• W2076123467 countsByYear W20761234672014 @default.
• W2076123467 countsByYear W20761234672015 @default.
• W2076123467 countsByYear W20761234672016 @default.
• W2076123467 countsByYear W20761234672017 @default.
• W2076123467 countsByYear W20761234672018 @default.
• W2076123467 countsByYear W20761234672019 @default.
• W2076123467 countsByYear W20761234672020 @default.
• W2076123467 countsByYear W20761234672021 @default.
• W2076123467 countsByYear W20761234672022 @default.
• W2076123467 countsByYear W20761234672023 @default.
• W2076123467 crossrefType "journal-article" @default.
• W2076123467 hasAuthorship W2076123467A5004705861 @default.
• W2076123467 hasAuthorship W2076123467A5008425789 @default.
• W2076123467 hasAuthorship W2076123467A5010311721 @default.
• W2076123467 hasAuthorship W2076123467A5012885531 @default.
• W2076123467 hasAuthorship W2076123467A5015904630 @default.
• W2076123467 hasAuthorship W2076123467A5016713193 @default.
• W2076123467 hasAuthorship W2076123467A5018959877 @default.
• W2076123467 hasAuthorship W2076123467A5040756867 @default.
• W2076123467 hasAuthorship W2076123467A5045419573 @default.
• W2076123467 hasAuthorship W2076123467A5049814810 @default.
• W2076123467 hasAuthorship W2076123467A5053464984 @default.
• W2076123467 hasAuthorship W2076123467A5077666402 @default.
• W2076123467 hasAuthorship W2076123467A5077884718 @default.
• W2076123467 hasAuthorship W2076123467A5083923366 @default.
• W2076123467 hasBestOaLocation W20761234672 @default.
• W2076123467 hasConcept C104317684 @default.
• W2076123467 hasConcept C126322002 @default.
• W2076123467 hasConcept C197934379 @default.
• W2076123467 hasConcept C203014093 @default.
• W2076123467 hasConcept C2777075537 @default.
• W2076123467 hasConcept C2777499176 @default.
• W2076123467 hasConcept C2778606649 @default.
• W2076123467 hasConcept C2779018223 @default.
• W2076123467 hasConcept C2779306644 @default.
• W2076123467 hasConcept C2779468605 @default.
• W2076123467 hasConcept C2780668416 @default.
• W2076123467 hasConcept C40767141 @default.
• W2076123467 hasConcept C55493867 @default.
• W2076123467 hasConcept C71924100 @default.
• W2076123467 hasConcept C86803240 @default.
• W2076123467 hasConcept C90924648 @default.
• W2076123467 hasConcept C92087593 @default.
• W2076123467 hasConceptScore W2076123467C104317684 @default.
• W2076123467 hasConceptScore W2076123467C126322002 @default.

• next