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Methods for exon skipping and gene knockout using base editors

  • Pablo Perez Pinera (Inventor)
  • , Michael P. Gapinske (Inventor)
  • , Jackson Scott Winter (Inventor)

Research output: Patent

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.
Original languageEnglish (US)
U.S. patent number12612612
Filing date6/19/19
StatePublished - Apr 28 2026

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