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Persistent in vivo epigenetic silencing of Pcsk9

Isabella R. Gengaro^1,2 , Lei S. Qi^2,3,4,*

¹Department of Chemical Engineering, Stanford University, Stanford, CA, USA
²Sarafan ChEM-H, Stanford University, Stanford, CA, USA
³Department of Bioengineering, Stanford University, Stanford, CA, USA
⁴Chan Zuckerberg Biohub–San Francisco, San Francisco, CA, USA
* Correspondence: Lei S. Qi(slqi@stanford.edu)

The quest for safer therapeutic alternatives to genome editing heralds epigenome editing as a promising approach. In a recent study published in Nature, Cappelluti et al. demonstrated robust, long-term Pcsk9 silencing in vivo with a single administration of epigenetic effectors delivered by lipid nanoparticles, marking a significant stride towards the clinic for epigenetic editing therapies.

https://doi.org/10.1038/s41422-024-00954-z

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