BioengineeringbioRxivPreprint
Engineering novel AAV capsids by global de-targeting and subsequent muscle-specific tropism in mice and NHPs
Pan Yue, Yujian Zhong, Huan Chen…·Huapeng Li
The paper develops AAV.eM, a muscle-specific rAAV capsid with reduced off-target transduction, showing efficacy in Duchenne Muscular Dystrophy models and favorable safety in mice and NHPs.
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