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Methods and products for nucleic acid production and delivery

  • US 9,770,489 B2
  • Filed: 01/30/2015
  • Issued: 09/26/2017
  • Est. Priority Date: 01/31/2014
  • Status: Active Grant
First Claim
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1. An in vivo method for treating dystrophic epidermolysis bullosa, comprisingdelivering a synthetic RNA encoding a gene-editing protein that targets a COL7 gene to a patient in need thereof anddelivering a COL7 repair template to the patient,thereby editing the COL7 gene, wherein:

  • the synthetic RNA and repair template are delivered to the patient'"'"'s keratinocytes by injection to the epidermis andthe gene-editing protein comprises a DNA-binding domain and a nuclease domain and causes a double-strand break in the COL7 gene of the patient'"'"'s keratinocytes.

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