Journal article
Sustained and therapeutic delivery of factor IX in nude haemophilia B mice by encapsulated C2C12 myoblasts: concurrent tumourigenesis
Abstract
This study reports the generation of an immunodeficient murine model for haemophilia B, obtained by breeding factor IX-deficient mice with an immunodeficient mouse strain, and use of this mouse model to evaluate the long-term efficacy and safety of a gene therapy strategy for treating haemophilia B. Nude haemophilic mice were implanted with biocompatible microcapsules enclosing recombinant myoblasts secreting human factor IX. The activated …
Authors
Hortelano G; Wang L; Xu N; Ofosu FA
Journal
Haemophilia, Vol. 7, No. 2, pp. 207–214
Publisher
Wiley
Publication Date
March 14, 2001
DOI
10.1046/j.1365-2516.2001.00492.x
ISSN
1351-8216