Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

Author:

Snyder Richard O.,Miao Carol H.,Patijn Gijsbert A.,Spratt S. Kaye,Danos Olivier,Nagy Dea,Gown Allen M.,Winther Brian,Meuse Leonard,Cohen Lawrence K.,Thompson Arthur R,Kay Mark A.

Publisher

Springer Science and Business Media LLC

Subject

Genetics

Reference36 articles.

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2. Kay, M.A. et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science 262, 117–119 (1993).

3. Kay, M.A. et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. USA 91, 2353–2357 (1994).

4. Yang, Y. et al. Cellular immunity to viral antigelns limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA 91, 4407–4411 (1994).

5. Muzyczka, N. Use of adeno-associated virus as a general transduction vector for mammalian cells. Curr. Top. Microbiol. Immunol. 158, 97–129 (1992).

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