Researchers published two genome‑editing advances: St. Jude Children’s Research Hospital reported CHANGE‑seq‑BE, an unbiased method to map base‑editor off‑target activity and support safety assessments for clinical applications; and a separate team demonstrated CRISPR–Cas3‑based long‑deletion editing targeting the TTR gene in a mouse model of transthyretin amyloidosis, achieving substantial hepatic editing and up to ~80% serum TTR reduction. CHANGE‑seq‑BE is already being used to supply specificity data to regulators; Cas3 work highlights an alternative editing mechanism that produces large directional deletions and may reduce in‑frame residual protein risk compared with nuclease‑indel approaches.
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