Programmable multi-kilobase RNA editing using CRISPR-mediated trans-splicing.

Current gene editing approaches in eukaryotic cells are limited to single base edits or small DNA insertions and deletions, and remain encumbered by unintended permanent effects and significant challenges in the delivery of large DNA cargo. Here we describe Splice Editing, a generalizable platform to correct gene transcripts

Déclaration de conflit d'intérêts

Competing interests: B.A-S and J.D.B are co-founders of Amber Bio. K.J., J.G., S.S., D.Y., K.H. are employees of Amber Bio. P.C.B. serves as a consultant to and equity holder in Amber Bio, and further consults with or holds equity in 10X Genomics, General Automation Lab Technologies/Isolation Bio, Celsius Therapeutics, Next Gen Diagnostics, Cache DNA, Concerto Biosciences, Stately, Ramona Optics, and Bifrost Biosystems, and his group receives industry funding for unrelated research. Amber Bio and MIT have filed patent applications on aspects of the work described in this manuscript, on which the authors are inventors. The Regents of the University of California have patents and patents pending on CRISPR technologies on which B.A-S is an inventor.