Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors
نویسندگان
چکیده
منابع مشابه
Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter.
Glycogen storage disease type II (Pompe disease) causes death in infancy from cardiorespiratory failure due to acid alpha-glucosidase (GAA; acid maltase) deficiency. An AAV2 vector pseudotyped as AAV6 (AAV2/6 vector) transiently expressed high-level human GAA in GAA-knockout (GAA-KO) mice without reducing glycogen storage; however, in immunodeficient GAA-KO/SCID mice the AAV2/6 vector expressed...
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SUMMARY The unique life cycle of adeno-associated virus (AAV) and its ability to infect both nondividing and dividing cells with persistent expression have made it an attractive vector. An additional attractive feature of the wild-type virus is the lack of apparent pathogenicity. Gene transfer studies using AAV have shown significant progress at the level of animal models; clinical trials have ...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2005
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3302550