title: A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo.
abstract: We have recently shown that co-administration of conventional single-stranded adeno-associated virus 2 (ssAAV2) vectors with self-complementary (sc) AAV2-protein phosphatase 5 (PP5) vectors leads to a significant increase in the transduction efficiency of ssAAV2 vectors in human cells in vitro as well as in murine hepatocytes in vivo. In the present study, this strategy has been further optimized by generating a mixed population of ssAAV2-EGFP and scAAV2-PP5 vectors at a 10:1 ratio to achieve enhanced green fluorescent protein (EGFP) transgene expression at approximately 5- to 10-fold higher efficiency, both in vitro and in vivo. This simple coproduction method should be adaptable to any ssAAV serotype vector containing transgene cassettes that are too large to be encapsidated in scAAV vectors.
authors.affiliation: Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA.
authors.forename: Wenqin
authors.initials: W
authors.lastname: Ma
authors.forename: Baozheng
authors.initials: B
authors.lastname: Li
authors.forename: Chen
authors.initials: C
authors.lastname: Ling
authors.forename: Giridhara R
authors.initials: GR
authors.lastname: Jayandharan
authors.forename: Arun
authors.initials: A
authors.lastname: Srivastava
authors.forename: Barry J
authors.initials: BJ
authors.lastname: Byrne
mesh_terms: D000818:Animals; D002460:Cell Line; D003001:Cloning, Molecular / Q000379:methods; D000229:Dependovirus; D005822:Genetic Vectors / Q000235:genetics*; D049452:Green Fluorescent Proteins / Q000378:metabolism; D022781:Hepatocytes / Q000378:metabolism; D006801:Humans; D066298:In Vitro Techniques; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D009687:Nuclear Proteins / Q000235:genetics; D010749:Phosphoprotein Phosphatases / Q000235:genetics; D010957:Plasmids / Q000235:genetics; D014161:Transduction, Genetic / Q000379:methods*
chemical_list: D009687:Nuclear Proteins; C485184:enhanced green fluorescent protein; D049452:Green Fluorescent Proteins; D010749:Phosphoprotein Phosphatases; C089641:protein phosphatase 5