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Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors.
University of Pennsylvania Health System, Institute for Human Gene Therapy, Philadelphia 19104, USA.
PMID: 10757357 [PubMed - indexed for MEDLINE]
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Cited by 3 PubMed Central articles
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Enhancing transduction of the liver by adeno-associated viral vectors.
Nathwani AC, Cochrane M, McIntosh J, Ng CY, Zhou J, Gray JT, Davidoff AM.
Gene Ther. 2009 Jan; 16(1):60-9. Epub 2008 Aug 14.
[Gene Ther. 2009]
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Proteolytic mapping of the adeno-associated virus capsid.
Van Vliet K, Blouin V, Agbandje-McKenna M, Snyder RO.
Mol Ther. 2006 Dec; 14(6):809-21. Epub 2006 Sep 27.
[Mol Ther. 2006]
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A novel gene expression control system and its use in stable, high-titer 293 cell-based adeno-associated virus packaging cell lines.
Qiao C, Wang B, Zhu X, Li J, Xiao X.
J Virol. 2002 Dec; 76(24):13015-27.
[J Virol. 2002]