Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

Author:

Chao Hengjun,Mansfield S Gary,Bartel Robert C,Hiriyanna Suja,Mitchell Lloyd G,Garcia-Blanco Mariano A,Walsh Christopher E

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Reference22 articles.

1. Kaufman, R.J. & Antonarakis, S.E. Structure, biology, and genetics of factor VIII. in Hematology: Basic Principles and Practice Vol. VIII-108 (ed. McGlave, P.) 1850–1868 (Church Livingstone, New York, 2000).

2. Lillicrap, D. Hemophilia treatment. Gene therapy, factor VIII antibodies and immune tolerance: hopes and concerns. Haematologica 85, 108–111 (2000).

3. Bi, L. et al. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat. Genet. 10, 119–121 (1995).

4. Bi, L. et al. Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies. Blood 88, 3446–3450 (1996).

5. Sullenger, B.A. & Cech, T.R. Ribozyme-mediated repair of defective mRNA by targeted, trans-splicing. Nature 371, 619–622 (1994).

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