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Genetic correction of mutated genes

  • US 10,711,256 B2
  • Filed: 06/27/2017
  • Issued: 07/14/2020
  • Est. Priority Date: 04/27/2012
  • Status: Active Grant
First Claim
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1. An engineered transcription activator-like effector nuclease (TALEN) protein that binds to a dystrophin gene, wherein the TALEN protein binds upstream from a premature stop codon on the dystrophin gene, downstream from a premature stop codon on the dystrophin gene, a region in exon 51 of the dystrophin gene, or a region in the 5′

  • UTR of the dystrophin gene, wherein the TALEN protein comprises an amino acid sequence selected from the group consisting of the amino acid sequence of SEQ ID NO;

    11, SEQ ID NO;

    12, SEQ ID NO;

    13, SEQ ID NO;

    14, and SEQ ID NO;

    15.

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