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Discover the latest scientific advancements and innovations in CRISPR-Cas9 technology, curated and summarized for you.

Schéma CRISPR-Cas3 induisant une large délétion sur le gène TTR pour traiter l’ATTR

CRISPR-Cas3 for ATTR: When “Deleting” Beats “Cutting”

Researchers from the University of Tokyo led by Tomoji Mashimo and Kazuto Yoshimi (IMS-UTokyo) have shown that CRISPR-Cas3 editing can inactivate the TTR gene via long, predominantly directional deletions, with robust efficacy in mouse liver and a highly favorable safety profile (off-target/IFM) compared to Cas9.

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