Source:http://linkedlifedata.com/resource/pubmed/id/14551134
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Predicate | Object |
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rdf:type | |
lifeskim:mentions | |
pubmed:issue |
4
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pubmed:dateCreated |
2004-2-3
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pubmed:abstractText |
Despite the popularity of adeno-associated virus 2 (AAV2) as a vehicle for gene transfer, its efficacy for liver-directed gene therapy in hemophilia A or B has been suboptimal. Here we evaluated AAV serotypes 2, 5, 7, and 8 in gene therapy of factor VIII (FVIII) deficiency in a hemophilia A mouse model and found that AAV8 was superior to the other 3 serotypes. We expressed canine B domain-deleted FVIII cDNA either in a single vector or in 2 separate AAV vectors containing the heavy- and light-chain cDNAs. We also evaluated AAV8 against AAV2 in intraportal and tail vein injections. AAV8 gave 100% correction of plasma FVIII activity irrespective of the vector type or route of administration.
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pubmed:language |
eng
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pubmed:journal | |
pubmed:citationSubset |
AIM
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pubmed:chemical | |
pubmed:status |
MEDLINE
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pubmed:month |
Feb
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pubmed:issn |
0006-4971
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pubmed:author | |
pubmed:issnType |
Print
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pubmed:day |
15
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pubmed:volume |
103
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
1253-60
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pubmed:dateRevised |
2006-11-15
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pubmed:meshHeading |
pubmed-meshheading:14551134-Adenoviridae,
pubmed-meshheading:14551134-Animals,
pubmed-meshheading:14551134-Blotting, Southern,
pubmed-meshheading:14551134-DNA, Recombinant,
pubmed-meshheading:14551134-Dogs,
pubmed-meshheading:14551134-Factor VIII,
pubmed-meshheading:14551134-Female,
pubmed-meshheading:14551134-Gene Therapy,
pubmed-meshheading:14551134-Genetic Vectors,
pubmed-meshheading:14551134-Hemophilia A,
pubmed-meshheading:14551134-Liver,
pubmed-meshheading:14551134-Male,
pubmed-meshheading:14551134-Mice,
pubmed-meshheading:14551134-Mice, Mutant Strains,
pubmed-meshheading:14551134-Portal Vein,
pubmed-meshheading:14551134-Tail
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pubmed:year |
2004
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pubmed:articleTitle |
Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.
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pubmed:affiliation |
Department of Genetics, University of Pennsylvania 415 Curie Blvd, CRB Rm 475, Philadelphia, PA 19104, USA. sarkarr@mail.med.upenn.edu
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pubmed:publicationType |
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Research Support, Non-U.S. Gov't
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