FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2149021748> ?p ?o ?g. }

• W2149021748 endingPage "327" @default.
• W2149021748 startingPage "317" @default.
• W2149021748 abstract "Summary We have generated an E1a/E1b/E3-deleted adeno/adeno-associated (Ad/AAV) hybrid virus driven by a small nuclear RNA (pHU1-1) promoter for expression of a B domain-deleted (Thr761-Asn1639) factor VIII transgene (FVIIIΔ761-1639). Productive replication of Ad/AAV/FVIIIΔ761-1639 in AAV repexpressing cells resulted in generation of monomeric and dimeric mini-adenoviral (mAd) replicative forms that retained the AAV integration elements (mAd/FVIIIΔ761-1639). In vitro studies using Ad/AAV/FVIIIΔ761-1639 generated ∼2-logs greater FVIII activity than mAd/FVIIIΔ761-1639. To determine its capacity for in vivo excision and/or genomic integration, Ad/AAV/FVIIIΔ761-1639 was injected by tail vein into three groups of hemophilia A mice (2 X 1011 vp [n = 3]; 4 X 1011 vp [n = 3]; 8 X 1011 vp [n = 3]), with clear concentration-dependent increase in FVIII activity (range 160-510 mU/ml; plasma activity 16% – 51% of normal). Peak activity was seen by Day (D) 5, with slow return to baseline by D28 (0.1 – 0.9% activity); in only 3/9 mice was loss of FVIII activity associated with development of anti-FVIII antibodies. Quantitative-PCR using genomic DNA isolated from D28 liver, spleen, heart, lungs, and kidney demonstrated the highest concentration in liver (∼10 genomes/ cell), with little to no organ toxicity at early (D5 or 6) or late (D28) post-infusion time points. There was no evidence for spontaneous transgene excision or genomic integration in vivo as evaluated by quantitative PCR and genomic blotting. These data establish (i) the feasibility and applicability of developing high-titer Ad/AAV hybrid viruses for FVIII delivery using a small cellular promoter, (ii) the potential utility of this virus for generation of “gutted” monomeric and dimeric mAD/FVIII retaining AAV integration elements, and (iii) that the development of strategies for regulated Rep68/78 co-expression may provide a novel approach for excision, integration, and long-term FVIII transgene expression." @default.
• W2149021748 created "2016-06-24" @default.
• W2149021748 creator A5014274785 @default.
• W2149021748 creator A5023967974 @default.
• W2149021748 creator A5030554078 @default.
• W2149021748 creator A5033649826 @default.
• W2149021748 creator A5063322136 @default.
• W2149021748 creator A5072840852 @default.
• W2149021748 date "2004-01-01" @default.
• W2149021748 modified "2023-09-26" @default.
• W2149021748 title "Expression of therapeutic levels of factor VIII in hemophilia A mice using a novel adeno/adeno-associated hybrid virus" @default.
• W2149021748 cites W1533835843 @default.
• W2149021748 cites W1541189153 @default.
• W2149021748 cites W1586940012 @default.
• W2149021748 cites W1594355808 @default.
• W2149021748 cites W1740655975 @default.
• W2149021748 cites W1859180288 @default.
• W2149021748 cites W1901002830 @default.
• W2149021748 cites W1964975805 @default.
• W2149021748 cites W1967446284 @default.
• W2149021748 cites W1986594968 @default.
• W2149021748 cites W2010126361 @default.
• W2149021748 cites W2023820753 @default.
• W2149021748 cites W2028043537 @default.
• W2149021748 cites W2034647149 @default.
• W2149021748 cites W2055599800 @default.
• W2149021748 cites W2069611627 @default.
• W2149021748 cites W2079017842 @default.
• W2149021748 cites W2102016263 @default.
• W2149021748 cites W2105056822 @default.
• W2149021748 cites W2115800975 @default.
• W2149021748 cites W2119164964 @default.
• W2149021748 cites W2120066089 @default.
• W2149021748 cites W2141296965 @default.
• W2149021748 cites W2142439096 @default.
• W2149021748 cites W2144373954 @default.
• W2149021748 cites W2151514148 @default.
• W2149021748 cites W2157466002 @default.
• W2149021748 cites W2158480664 @default.
• W2149021748 cites W2165615022 @default.
• W2149021748 cites W2167551295 @default.
• W2149021748 cites W2243590771 @default.
• W2149021748 cites W2312320520 @default.
• W2149021748 cites W32161039 @default.
• W2149021748 cites W2084017165 @default.
• W2149021748 cites W2470164098 @default.
• W2149021748 doi "https://doi.org/10.1160/th04-02-0068" @default.
• W2149021748 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/15269828" @default.
• W2149021748 hasPublicationYear "2004" @default.
• W2149021748 type Work @default.
• W2149021748 sameAs 2149021748 @default.
• W2149021748 citedByCount "8" @default.
• W2149021748 countsByYear W21490217482022 @default.
• W2149021748 crossrefType "journal-article" @default.
• W2149021748 hasAuthorship W2149021748A5014274785 @default.
• W2149021748 hasAuthorship W2149021748A5023967974 @default.
• W2149021748 hasAuthorship W2149021748A5030554078 @default.
• W2149021748 hasAuthorship W2149021748A5033649826 @default.
• W2149021748 hasAuthorship W2149021748A5063322136 @default.
• W2149021748 hasAuthorship W2149021748A5072840852 @default.
• W2149021748 hasConcept C102230213 @default.
• W2149021748 hasConcept C104317684 @default.
• W2149021748 hasConcept C141035611 @default.
• W2149021748 hasConcept C153911025 @default.
• W2149021748 hasConcept C159047783 @default.
• W2149021748 hasConcept C202751555 @default.
• W2149021748 hasConcept C203014093 @default.
• W2149021748 hasConcept C207001950 @default.
• W2149021748 hasConcept C2522874641 @default.
• W2149021748 hasConcept C2778107364 @default.
• W2149021748 hasConcept C2780931953 @default.
• W2149021748 hasConcept C40767141 @default.
• W2149021748 hasConcept C54355233 @default.
• W2149021748 hasConcept C86803240 @default.
• W2149021748 hasConcept C92087593 @default.
• W2149021748 hasConceptScore W2149021748C102230213 @default.
• W2149021748 hasConceptScore W2149021748C104317684 @default.
• W2149021748 hasConceptScore W2149021748C141035611 @default.
• W2149021748 hasConceptScore W2149021748C153911025 @default.
• W2149021748 hasConceptScore W2149021748C159047783 @default.
• W2149021748 hasConceptScore W2149021748C202751555 @default.
• W2149021748 hasConceptScore W2149021748C203014093 @default.
• W2149021748 hasConceptScore W2149021748C207001950 @default.
• W2149021748 hasConceptScore W2149021748C2522874641 @default.
• W2149021748 hasConceptScore W2149021748C2778107364 @default.
• W2149021748 hasConceptScore W2149021748C2780931953 @default.
• W2149021748 hasConceptScore W2149021748C40767141 @default.
• W2149021748 hasConceptScore W2149021748C54355233 @default.
• W2149021748 hasConceptScore W2149021748C86803240 @default.
• W2149021748 hasConceptScore W2149021748C92087593 @default.
• W2149021748 hasFunder F4320332161 @default.
• W2149021748 hasIssue "08" @default.
• W2149021748 hasLocation W21490217481 @default.
• W2149021748 hasLocation W21490217482 @default.
• W2149021748 hasOpenAccess W2149021748 @default.
• W2149021748 hasPrimaryLocation W21490217481 @default.
• W2149021748 hasRelatedWork W1024660077 @default.
• W2149021748 hasRelatedWork W1933273068 @default.

• next