Preclinical study demonstrates efficient generation of disease models in mice, indicating expanded capability to model complex mutations beyond standard base editors.
Most genetic diseases in humans are caused by single-nucleotide mutations. Although genome editing with either the CRISPR-based cytosine base editor (CBE) 1 or the adenine base editor (ABE) 2 holds great promise for gene correction of C-to-T and A-to-G base substitutions in some genetic diseases 3 , 4 , both editors are useless for correction of other variants such as base transversion, small insertions and deletions (indels).
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Liu et al. (2020) studied this question.
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