期刊
TRANSLATIONAL RESEARCH
卷 161, 期 4, 页码 307-312出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/j.trsl.2012.12.016
关键词
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资金
- Hemophilia Consortium
- Fanconi Anemia Research Fund
Genetic correction of hemophilia A and B was long considered amenable to the available gene transfer technologies. This assumption has come to fruition with the recent results of a phase I/II trial for hemophilia B. Here we review the clinical application of gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer science and technology. This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias. (Translational Research 2013;161:307-312)
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