Modified polynucleotides for treating galactosylceramidase protein deficiency
AssigneeModerna Therapeutics, Inc.
InventorsTirtha Chakraborty, Antonin de Fougerolles, Ron Weiss
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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.
Claims16
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.
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.
Dependencies are inferred from the claim text.