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Methods for correcting presenilin point mutations

  • US 9,068,179 B1
  • Filed: 07/08/2014
  • Issued: 06/30/2015
  • Est. Priority Date: 12/12/2013
  • Status: Active Grant
First Claim
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1. A method of editing a nucleic acid molecule encoding a Presenilin1 (PSEN1) protein, the method comprising contacting the nucleic acid molecule with(a) a fusion protein comprising a nuclease-inactive Cas9 domain and a deaminase domain;

  • and(b) a single guide RNA (sgRNA) targeting the fusion protein of (a) to the PSEN1-encoding nucleic acid molecule;

    wherein the nucleic acid molecule comprises a T>

    C and/or an A>

    G point mutation in the PSEN1-encoding nucleic acid molecule as compared to a wild-type PSEN1-encoding nucleic acid molecule,and wherein the PSEN1-encoding nucleic acid molecule is contacted with the fusion protein and the sgRNA in an amount effective and under conditions suitable for the deamination of the mutant C or G nucleotide base.

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