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US 12,612,612 B2Granted
Methods for exon skipping and gene knockout using base editors
AssigneeThe Board of Trustees of the University of Illinois
InventorsPablo Perez-Pinera, Michael P. Gapinske, Jackson Scott Winter
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Abstract
The disclosure provides a versatile method termed CRISPR-SKIP that utilizes cytidine and/or adenine deaminase base editors to program exon skipping by mutating target DNA bases within splice acceptor sites and/or splice enhancer sites. Given its simplicity and precision, CRISPR-SKIP will be broadly applicable in gene therapy and synthetic biology.
Claims14
A method for inducing selective exon skipping comprising:
- contacting one or more DNA target sequences with (i) a single guide RNA (sgRNA) molecule having complementarity to the one or more DNA target sequences;
- and (ii) a fusion protein comprising (i) at least two tRNA-specific adenosine deaminases (TadA) domains (ii) a linker;
- and (iii) an RNA-guided DNA endonuclease having nickase activity capable of causing single stranded breaks in the one or more DNA target sequences, such that an exon is selectively skipped.
Dependencies are inferred from the claim text.