Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer.
Vectors based on hybrids consisting of adeno-associated virus types 2 (ITRs and Rep) and 5 (Cap) were evaluated for muscle-directed gene transfer (called AAV2/5). Evaluation in immune-competent mice revealed greater transduction efficacy with AAV2/5 than with AAV2 and no cross-neutralization between AAV2/5 and AAV2. Interestingly, we saw no immunologic evidence of previous exposure to AAV5 capsids in a large population of healthy human subjects.
Hildinger
M
M
Institute for Human Gene Therapy, Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Auricchio
A
A
Gao
G
G
Wang
L
L
Chirmule
N
N
Wilson
J M
JM
D000818:Animals; D000914:Antibodies, Viral / Q000097:blood; D000229:Dependovirus / Q000145:classification / Q000235:genetics* / Q000276:immunology; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D006824:Hybridization, Genetic; D007114:Immunization; D051379:Mice; D008810:Mice, Inbred C57BL; D009132:Muscles / Q000378:metabolism*; D012703:Serotyping
D000914:Antibodies, Viral