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. 2014 Aug 13;1:14033. doi: 10.1038/mtm.2014.33

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Copyright © 2014 American Society of Gene & Cell Therapy

This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivs 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-nc-nd/3.0/

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A 3.5 year-old Pompe disease subject (solid lines, diamonds) received weekly administration of 375 mg/m² of rituximab along with 10 mg/kg of methylprednisolone for 3 weeks prior to dosing with 5 × 10¹² vg/kg of rAAV1-CMV-hGAA. The patient then received daily oral administration of 0.06–1 mg/m² of Sirolimus through the study and continued B-cell depletion with Rituximab every 12 weeks. Five Pompe subjects (dash-dot lines, circles), age 2.5–15 years, received 1–5 × 10¹² vg/kg of rAAV1-CMV-GAA without immune modulation, served as a control group. The light horizontal lines represent +2 SD above the population mean. (a) Circulating antibodies against AAV1. (b) Circulating antibodies against hGAA. (c) Antigen specific response assay versus AAV1. (d) Antigen specific response assay versus hGAA.