Recombinant adeno-associated virus vectors for gene therapy.
Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA, activation of transcription and establishment of persistent molecular forms. Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials.
Conlon
Thomas J
TJ
University of Florida College of Medicine, Department of Pediatrics, Box 100296, Gainesville, FL 32610-0296, USA.
Flotte
Terence R
TR
D000818:Animals; D001692:Biological Transport; D002213:Capsid / Q000648:ultrastructure; D002986:Clinical Trials as Topic; D004274:DNA, Recombinant / Q000235:genetics; D004277:DNA, Single-Stranded / Q000235:genetics; D004279:DNA, Viral / Q000235:genetics; D000229:Dependovirus / Q000235:genetics* / Q000472:pathogenicity / Q000648:ultrastructure; D015967:Gene Expression Regulation, Viral; D005813:Genes, Synthetic; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics / Q000627:therapeutic use*; D006801:Humans; D007273:Injections, Intramuscular; D018485:Muscle Fibers, Skeletal / Q000821:virology; D016254:Mutagenesis, Insertional; D011991:Receptors, Virus / Q000502:physiology; D014161:Transduction, Genetic
D004274:DNA, Recombinant; D004277:DNA, Single-Stranded; D004279:DNA, Viral; D011991:Receptors, Virus