Progress in the use of adeno-associated viral vectors for gene therapy

The development of safe and efficient gene transfer vectors is
crucial for the success of gene therapy trials. A viral vector
system promising to meet these requirements is based on the
apathogenic adeno-associated virus (AAV-2), a member of the
parvovirus family. The advantages of this vector system is the
stability of the viral capsid, the low immunogenicity, the ability
to transduce both dividing and non-dividing cells, the potential to
integrate site specifically and to achieve long-term gene
expression even in vivo, and its broad tropism allowing the
efficient transduction of diverse organs including the skin. All
this makes AAV-2 attractive and efficient for in vitro gene
transfer and local injection in vivo. This review covers the
progress made in AAV vector technology including the development of
AAV vectors based on other serotypes, summarizes the results
obtained by AAV targeting vectors and outlines potential
applications in the field of cutaneous gene therapy. Copyright (C)
2004 S. Karger AG, Basel.