BioengineeringMolecular TherapyPeer-reviewed
Treatment of Huntington's disease with a pan-HTT-targeting CRISPR nuclease
Katherine Tan, Daniela Del Bosque Siller, Alisha Y. Xiong…·Thomas Gaj
This study develops a CRISPR-Cas9 system targeting all HTT alleles in HD mouse models, reducing mutant HTT and improving disease symptoms.
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