ArticleNature biomedical engineering2026
Template-independent genome editing and restoration for correcting frameshift disorders.
Article in Nature biomedical engineering, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Authors and funding
16 authors.
Funding
Abstract
Frameshift mutations, responsible for >20% of Mendelian inherited diseases, pose substantial therapeutic challenges. Here we developed Template-Independent Genome Editing for Restoration (TIGER), a platform for the efficient and precise correction of frameshift mutations across various models. By identifying reproducible nucleotide-level factors that influence therapeutic efficacy across cells and tissues, we developed a scoring system for guide RNA (gRNA)-Cas9 outcomes. Approximately 75% of deletion and 50% of insertion mutations produced ≥30% in-frame products, sufficient for phenotypic restoration, with 38% and 65% achieving wild-type correction, respectively. To expand the applicability of TIGER across species and genome wide, we retrained the inDelphi algorithm to predict therapeutic gRNAs for single-nucleotide frameshifts. In a mouse model of deafness, delivery of SpCas9 and optimal gRNA via dual adeno-associated virus restored hearing thresholds to wild-type levels, with ~90% of in-frame edits being wild type. TIGER provides a robust and broadly applicable strategy for in vivo correction of inherited frameshift diseases.
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Registered trials
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