Modified glucosidase, beta, acid polynucleotides for treating protein deficiency
AssigneeModerna Threrapeutics, Inc.
InventorsStephane Bancel, Tirtha Chakraborty, Antonin de Fougerolles, Susan Whoriskey
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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.
Claims9
A method of producing a polypeptide of interest in a mammalian cell or tissue comprising contacting said mammalian cell or tissue with a modified mRNA, wherein said modified mRNA encodes a member selected from the group consisting of glucosidase, beta, acid (SEQ ID NO:
- 1016), glucosidase, beta, acid (SEQ ID NO:
- 1017), glucosidase, beta, acid (SEQ ID NO:
- 1018), glucosidase, beta, acid (SEQ ID NO:
- 1019), glucosidase, beta, acid (SEQ ID NO:
- 1020), glucosidase, beta, acid (SEQ ID NO:
- 1021) and glucosidase, beta, acid (SEQ ID NO:
- 1022), and wherein said modified mRNA comprises modifications selected from the group consisting of:
- (a) 5-methylcytosine and 2-thiouridine,
- (b) pseudouridine and 5-methylcytosine, and
- (c) 1-methylpseudouridine.
A modified mRNA encoding a polypeptide of interest, said polypeptide of interest selected from the group consisting of glucosidase, beta, acid (SEQ ID NO:
- 1016), glucosidase, beta, acid (SEQ ID NO:
- 1017), glucosidase, beta, acid (SEQ ID NO:
- 1018), glucosidase, beta, acid (SEQ ID NO:
- 1019), glucosidase, beta, acid (SEQ ID NO:
- 1020), glucosidase, beta, acid (SEQ ID NO:
- 1021) and glucosidase, beta, acid (SEQ ID NO:
- 1022), and wherein said modified mRNA comprises modifications selected from the group consisting of:
- (a) 5-methylcytosine and 2-thiouridine,
- (b) pseudouridine and 5-methylcytosine, and
- (c) 1-methylpseudouridine.
(canceled)
Dependencies are inferred from the claim text.