Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response-Reference-Cited by-同舟云学术

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

Published:2006-02-12 Issue:3 Volume:12 Page:342-347
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

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

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/nm1358.pdf

Reference29 articles.

1. Mount, J.D. et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99, 2670–2676 (2002).

2. Ljung, R.C. Can haemophilic arthropathy be prevented? Br. J. Haematol. 101, 215–219 (1998).

3. Lofqvist, T., Nilsson, I.M., Berntorp, E. & Pettersson, H. Haemophilia prophylaxis in young patients–a long-term follow-up. J. Intern. Med. 241, 395–400 (1997).

4. Snyder, R.O. et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat. Med. 5, 64–70 (1999).

5. Wang, L., Nichols, T.C., Read, M.S., Bellinger, D.A. & Verma, I.M. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol. Ther. 1, 154–158 (2000).

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