Shandong Normal University

Jun Zhou

Songbo Xie

The present invention belongs to the technical field of proteolysis-targeting chimera preparation and biomedical technology, and specifically relates to a FUS proteolysis-targeting chimera, preparation method and applications thereof. Specifically, the present invention designed ligands specifically binding to FUS based on the 'GGUG' RNA motif that specifically binds to FUS, and developed FUS proteolysis-targeting chimeras that specifically degrade wild-type and mutant FUS by connecting CRBN ligands at their 5' end. Meanwhile, to ensure effective action of FUS proteolysis-targeting chimeras in the brain, the present invention designed DNA nanoflowers based on transferrin receptor aptamers, which can effectively load FUS proteolysis-targeting chimeras and deliver them to the brain for action, achieving precise medicine for ALS patients with FUS mutations.

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Jinan, China

Jun Zhou