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. 2014 Aug 6;1:14036. doi: 10.1038/mtm.2014.36

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Copyright © 2014 American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution 3.0 Unported License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/3.0/

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Dose finding of rAAV-HCR-ET3 in a murine model of hemophilia A. Dose finding studies were performed in male and female immune-competent hemophilia A mice. fVIII activity is presented from mice that did not form neutralizing antibodies. In male mice, (a) a long term, high dose study; (b) a medium duration, mid-dose extension study; and (c) a short term, minimum effective dose study were performed. In female mice, (d) a shorter duration dose finding study was performed. (e) A comparison between male and female mice receiving 4e12 vg/kg shows the intersex difference in fVIII levels. Baseline determination of fVIII activity in all mice tested was below the limit of detection of the assay (data not shown). N = 2–4 for all doses, error bars show one sample standard deviation. To determine the specific activity of in vivo rAAV expressed ET3, a panel of plasmas from rAAV-HCR-ET3 treated mice (open circles) were assayed for ET3 antigen level by ELISA and fVIII activity by chromogenic substrate activity assay. (f) The values obtained for each plasma were compared to a standard curve generated using highly purified recombinant ET3 diluted in hemophilia A mouse plasma (closed circles).