Modified polynucleotides for treating galactosylceramidase protein deficiency
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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Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
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Citations
16 Claims
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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. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8)
- untranslated region and a 5′
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9. An mRNA encoding a polypeptide of interest, said polypeptide of interest 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), said mRNA having a 3′
untranslated region and a 5′
untranslated region and wherein said mRNA comprises a coding region, said coding region having at least 85% identity to SEQ ID NO;
82603. - View Dependent Claims (10, 11, 12, 13, 14, 15, 16)
- 47205), galactosylceramidase (SEQ ID NO;
Specification