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Modified polynucleotides for treating galactosylceramidase protein deficiency

  • US 9,089,604 B2
  • Filed: 02/03/2014
  • Issued: 07/28/2015
  • Est. Priority Date: 04/02/2012
  • Status: Active Grant
First Claim
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1. A method of producing a polypeptide of interest in a mammalian cell or tissue comprising contacting said mammalian cell or tissue with an mRNA having a 3′

  • untranslated region and a 5′

    untranslated region, wherein said modified mRNA encodes a member selected from the group consisting of galactosylceramidase (SEQ ID NO;

         47205), galactosylceramidase (SEQ ID NO;

         47206), galactosylceramidase (SEQ ID NO;

         47207), galactosylceramidase (SEQ ID NO;

         47208) and galactosylceramidase (SEQ ID NO;

         47209), and wherein said mRNA comprises a coding region, said coding region having at least 85% identity to SEQ ID NO;

    82603.

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