Molecular Therapy

TABLE 1

FROM:

Helper-Independent Sleeping Beauty Transposon–Transposase Vectors for Efficient Nonviral Gene Delivery and Persistent Gene Expression in Vivo

Jacob Giehm Mikkelsen, Stephen R. Yant, Leonard Meuse, Zan Huang, Hui Xu and Mark A. Kay

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Table 1. Partial phenotypic correction in hemophilia B mice treated once with HITT-vector plasmids

GroupMouse strainVector treatmentMousehFIX (ng/ml) Day 110Bleeding times (min)
1C57Bl/6No DNA injection1ND14
   2ND13
   3ND10
2Hemophilia42 mug pT/EF1alpha-hFIX.PGK-SB432220
 C57Bl/6"active transposase"518718
   630319
   722122
3Hemophilia42 mug pT/EF1alpha-hFIX.PGK-mSB8UD>30
 C57Bl/6"inactive transposase"9UD>30
   10UD>30
   11UD>30
   12UD>30

 ND, not done; UD, undetectable by ELISA.

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