ABSTRACT
Presently, human gene therapy is limited by the efficiency of stable gene transfer. To overcome this obstacle, adeno-associated virus (AAV) is being developed as a vector. Since the initial description of recombinant AAV (rAAV) as a vector for gene transfer, substantial progress has been made towards the ultimate goal of using this vector in human gene therapy. Retroviral vectors based on the murine leukaemia virus require cell division for efficient transduction. A potential barrier to further in vivo studies with rAAV vectors has been the low transduction efficiencies of these vectors in some cell types. The impact of these findings on the use of AAV vectors for gene therapy is unclear. However, it is apparent that rAAV vector transduction can be improved dramatically in cultured cells by a number of physical and chemical manipulations. rAAV vectors are among the newest gene transfer vehicles.
