Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2006-03

AUTHORS

Catherine S Manno, Glenn F Pierce, Valder R Arruda, Bertil Glader, Margaret Ragni, John J E Rasko, Margareth C Ozelo, Keith Hoots, Philip Blatt, Barbara Konkle, Michael Dake, Robin Kaye, Mahmood Razavi, Albert Zajko, James Zehnder, Pradip Rustagi, Hiroyuki Nakai, Amy Chew, Debra Leonard, J Fraser Wright, Ruth R Lessard, Jürg M Sommer, Michael Tigges, Denise Sabatino, Alvin Luk, Haiyan Jiang, Federico Mingozzi, Linda Couto, Hildegund C Ertl, Katherine A High, Mark A Kay

ABSTRACT

We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of therapeutic levels of F.IX in dogs with severe hemophilia B. We carried out a phase 1/2 dose-escalation clinical study to extend this approach to humans with severe hemophilia B. rAAV-2 vector expressing human F.IX was infused through the hepatic artery into seven subjects. The data show that: (i) vector infusion at doses up to 2 x 10(12) vg/kg was not associated with acute or long-lasting toxicity; (ii) therapeutic levels of F.IX were achieved at the highest dose tested; (iii) duration of expression at therapeutic levels was limited to a period of approximately 8 weeks; (iv) a gradual decline in F.IX was accompanied by a transient asymptomatic elevation of liver transaminases that resolved without treatment. Further studies suggested that destruction of transduced hepatocytes by cell-mediated immunity targeting antigens of the AAV capsid caused both the decline in F.IX and the transient transaminitis. We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression. More... »

PAGES

342-347

Journal

TITLE

Nature Medicine

ISSUE

3

VOLUME

12

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  • Identifiers

    URI

    http://scigraph.springernature.com/pub.10.1038/nm1358

    DOI

    http://dx.doi.org/10.1038/nm1358

    DIMENSIONS

    https://app.dimensions.ai/details/publication/pub.1012134968

    PUBMED

    https://www.ncbi.nlm.nih.gov/pubmed/16474400


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