Researchers led by Professor Akitsu Hotta developed a CRISPR safety-evaluation framework aimed at assessing both intended and unintended mutations after gene editing delivered via lipid nanoparticles. The approach combines computational prediction, experimental validation, and whole-genome analysis. The work, published in Molecular Therapy – Nucleic Acids, is described as a practical strategy for improving the safety readout of genome-editing therapies by measuring off-target and broader genomic effects. By targeting a workflow that spans in silico design through in vivo-style validation and genome-wide profiling, the framework seeks to make safety assessment more systematic for next-generation CRISPR programs.
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