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METHODS FOR CORRECTING ALPHA-ANTITRYPSIN POINT MUTATIONS

  • US 20150166984A1
  • Filed: 07/08/2014
  • Published: 06/18/2015
  • Est. Priority Date: 12/12/2013
  • Status: Abandoned Application
First Claim
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1. A method of editing a nucleic acid molecule encoding an α

  • -antitrypsin 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) an sgRNA targeting the fusion protein of (a) to the α

    -antitrypsin-encoding nucleic acid molecule;

    wherein the nucleic acid molecule comprises a T>

    C and/or an A>

    G point mutation in the α

    -antitrypsin-encoding nucleic acid molecule as compared to a wild-type α

    -antitrypsin-encoding nucleic acid molecule,and wherein the α

    -antitrypsin-encoding nucleic acid molecule is contacted with the fusion protein and the sgRNA in an amount effective and under conditions suitable for the deamination the mutant C or G nucleotide base.

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