Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

Author:

Bunting Stuart,Zhang Lening,Xie Lin,Bullens Sherry,Mahimkar Rajeev,Fong Sylvia,Sandza Krystal,Harmon Danielle,Yates Bridget,Handyside Britta,Sihn Choong-Ryoul,Galicia Nicole,Tsuruda Laurie,O’Neill Charles A.,Bagri Anil,Colosi Peter,Long Shinong,Vehar Gordon,Carter Barrie

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference57 articles.

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3. Haemophilias A and B;Bolton-Maggs;Lancet,2003

4. A study of variations in the reported haemophilia A prevalence around the world;Stonebraker;Haemophilia,2010

5. Emerging and future therapies for hemophilia;Carr;J. Blood Med.,2015

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