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.
Büning
Hildegard
H
Laboratorium für Molekulare Biologie, Genzentrum, Ludwig-Maximilians-Universität München, Germany.
Braun-Falco
Markus
M
Hallek
Michael
M
D000818:Animals; D002455:Cell Division; D000229:Dependovirus / Q000145:classification / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors; D006801:Humans; D066298:In Vitro Techniques; D015603:Keratinocytes / Q000166:cytology / Q000378:metabolism / Q000821:virology; D009928:Organ Specificity; D011994:Recombinant Proteins / Q000096:biosynthesis / Q000235:genetics; D012703:Serotyping; D012871:Skin Diseases / Q000235:genetics / Q000628:therapy; D014161:Transduction, Genetic
D011994:Recombinant Proteins