发明申请
US20040192599A1 Gene therapy for hemophilia a 审中-公开
血友病基因治疗

Gene therapy for hemophilia a
摘要:
Nucleic acid constructs comprising procoagulant gene sequences under the control of a megakaryocyte-specific promoter are provided. The sequences preferably also comprise a secretory granule sorting domain Also provided are vectors comprising the sequences and methods of gene therapy comprising the use of the various constructs.
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