3.2. Experimental gene therapy

MF Marco Frentsch
AJ Alberto Sada Japp
MD Manuela Dingeldey
NM Nadine Matzmohr
AT Andreas Thiel
FS Friedrich Scheiflinger
BR Birgit M. Reipert
MR Maurus de la Rosa
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Mice were treated with two doses (5 × 1011 vector genomes [vg] per kg of body weight and 4 × 1012 vg/kg) of AAV8‐FIX or buffer. The groups received 500 μg abatacept (Bristol‐Myers Squibb) intraperitoneally 3 days and 1 day before injection of AAV8 vectors, and at days 1, 3, and 6 after injection. AAV8‐FIX was further injected with and without abatacept in the same way. Blood was collected from the tail at the indicated time points. Spleens were harvested on day 77.

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