166. Induction of immune tolerance and phenotypic rescue of hemophilic mice by stem cell mediated gene transfer

Brian W. Bigger,Elena K. Siapati,Ajay Mistry,Simon N. Waddington,Megha S. Nivsarkar,Maxine V. Holder,Chris Ridler,Geoff Kemball-Cook,Robyn R. Ali,Stuart J Forbes,Charles Coutelle,Nick Wright,Malcolm R Alison,Adrian J Thrasher,Dominique Bonnet,Michael Themis

Molecular Therapy（2004）

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摘要

Immune responses against an introduced transgene are a potential risk in many gene replacement strategies to treat genetic disease. Thus we have developed a gene delivery approach for hemophilia B based on lentiviral expression of human factor IX in purified hematopoietic stem cells.

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mt, INSERT KEY WORDS HERE, pharmacology

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