Source:http://linkedlifedata.com/resource/pubmed/id/16005263
Switch to
| Predicate | Object |
|---|---|
| rdf:type | |
| lifeskim:mentions | |
| pubmed:issue |
5
|
| pubmed:dateCreated |
2005-10-31
|
| pubmed:abstractText |
Glycogen storage disease type II (GSD-II; Pompe disease) is caused by a deficiency of acid alpha-glucosidase (GAA; acid maltase) and manifests as muscle weakness, hypertrophic cardiomyopathy, and respiratory failure. Adeno-associated virus vectors containing either a liver-specific promoter (LSP) (AAV-LSPhGAApA) or a hybrid CB promoter (AAV-CBhGAApA) to drive human GAA expression were pseudotyped as AAV8 and administered to immunocompetent GAA-knockout mice. Secreted hGAA was detectable in plasma between 1 day and 12 weeks postadministration with AAV-LSPhGAApA and only from 1 to 8 days postadministration for AAV-CBGAApA. No anti-GAA antibodies were detected in response to AAV-LSPhGAApA (<1:200), whereas AAV-CBhGAApA provoked an escalating antibody response starting 2 weeks postadministration. The LSP drove approximately 60-fold higher GAA expression than the CB promoter in the liver by 12 weeks following vector administration. Furthermore, the detected cellular immunity was provoked by AAV-CBhGAApA, as detected by ELISpot and CD4+/CD8+ lymphocyte immunodetection. GAA activity was increased to higher than normal and glycogen content was reduced to essentially normal levels in the heart and skeletal muscle following administration of AAV-LSPhGAApA. Therefore, liver-restricted GAA expression with an AAV vector evaded immunity and enhanced efficacy in GSD-II mice.
|
| pubmed:grant | |
| pubmed:language |
eng
|
| pubmed:journal | |
| pubmed:citationSubset |
IM
|
| pubmed:chemical |
http://linkedlifedata.com/resource/pubmed/chemical/Creatine Kinase,
http://linkedlifedata.com/resource/pubmed/chemical/Creatine Kinase, MM Form,
http://linkedlifedata.com/resource/pubmed/chemical/DNA, Viral,
http://linkedlifedata.com/resource/pubmed/chemical/Glycogen,
http://linkedlifedata.com/resource/pubmed/chemical/alpha-Glucosidases
|
| pubmed:status |
MEDLINE
|
| pubmed:month |
Nov
|
| pubmed:issn |
1525-0016
|
| pubmed:author |
pubmed-author:AmalfitanoAndreaA,
pubmed-author:BirdAndrewA,
pubmed-author:BrownTalmageT,
pubmed-author:ChenY TYT,
pubmed-author:ClayTimothy MTM,
pubmed-author:FrancoLuis MLM,
pubmed-author:KoeberlDwight DDD,
pubmed-author:SchneiderAynA,
pubmed-author:SunBaodongB,
pubmed-author:YangXiaoyiX,
pubmed-author:YoungSarah PSP,
pubmed-author:ZhangHaoyueH
|
| pubmed:issnType |
Print
|
| pubmed:volume |
12
|
| pubmed:owner |
NLM
|
| pubmed:authorsComplete |
Y
|
| pubmed:pagination |
876-84
|
| pubmed:dateRevised |
2008-11-21
|
| pubmed:meshHeading |
pubmed-meshheading:16005263-Animals,
pubmed-meshheading:16005263-Antibody Formation,
pubmed-meshheading:16005263-Creatine Kinase,
pubmed-meshheading:16005263-Creatine Kinase, MM Form,
pubmed-meshheading:16005263-DNA, Viral,
pubmed-meshheading:16005263-Dependovirus,
pubmed-meshheading:16005263-Enhancer Elements, Genetic,
pubmed-meshheading:16005263-Gene Therapy,
pubmed-meshheading:16005263-Gene Transfer Techniques,
pubmed-meshheading:16005263-Genetic Vectors,
pubmed-meshheading:16005263-Glycogen,
pubmed-meshheading:16005263-Glycogen Storage Disease Type II,
pubmed-meshheading:16005263-Humans,
pubmed-meshheading:16005263-Liver,
pubmed-meshheading:16005263-Mice,
pubmed-meshheading:16005263-Mice, Knockout,
pubmed-meshheading:16005263-Muscle, Skeletal,
pubmed-meshheading:16005263-Plasmids,
pubmed-meshheading:16005263-Promoter Regions, Genetic,
pubmed-meshheading:16005263-alpha-Glucosidases
|
| pubmed:year |
2005
|
| pubmed:articleTitle |
Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.
|
| pubmed:affiliation |
Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
|
| pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't,
Research Support, N.I.H., Extramural
|