Efficient in vivo gene transfer to xenotransplanted human skin by lentivirus-mediated, but not by AAV-directed, gene delivery

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Skin is an easily accessible organ, and therapeutic gene transfer to skin remains an attractive alternative for treatment of skin diseases. We compared the potential usefulness of various serotypes of recombinant AAV vectors and lentiviral vectors for gene transfer to human skin in a xenotransplanted mouse model. Vector constructs encoding firefly luciferase were packaged in AAV capsids of serotype 1, 2, 5, 6, 8, and 9 and separately administered by intradermal injection in human skin transplants. For all serotypes, live bioimaging
demonstrated low levels of transgene expression in the human skin graft, and firefly luciferase expression was observed primarily in neighboring tissue beneath or surrounding the graft. In contrast, gene delivery by intradermally injected lentiviral vectors was efficient and led to extensive and persistent firefly luciferase expression within the human skin graft only. The study demonstrates limited capacity of single-stranded AAV vectors of six commonly used serotypes for gene delivery to human skin in vivo.
Original languageEnglish
Publication year18 Sep 2015
Number of pages1
Publication statusPublished - 18 Sep 2015
EventAnnual Congress The European Society of Gene and Cell Therapy (ESGCT) - Finlandia Hall, Helsinki, Finland
Duration: 17 Sep 201520 Sep 2015


ConferenceAnnual Congress The European Society of Gene and Cell Therapy (ESGCT)
LocationFinlandia Hall

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