Adeno-associated virus vectors for gene therapy: more pros than cons?
Gene therapy vectors based on the adeno-associated virus (AAV) are being developed for a widening variety of therapeutic applications. Enthusiasm for AAV is due, not only to the relative safety of these vectors, but also to advances in understanding of the unique biology of this virus. This review examines a number of long-standing concerns regarding the utility of AAV for gene transfer in light of many new insights into the biology, immunology and production of AAV.
Monahan
P E
PE
Division of Hematology/Oncology and Gene Therapy Center, University of North Carolina at Chapel Hill School of Medicine, CB#7352, Thurston-Bowles Bldg, Chapel Hill, NC 27599, USA. Paul_Monahan@med.unc.edu
Samulski
R J
RJ
D000229:Dependovirus / Q000235:genetics* / Q000276:immunology / Q000502:physiology; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D016297:Mutagenesis, Site-Directed / Q000235:genetics; D009928:Organ Specificity; D014161:Transduction, Genetic; D019076:Transgenes / Q000235:genetics; D019065:Virus Assembly / Q000502:physiology; D016662:Virus Integration / Q000235:genetics