Modified polynucleotides for treating dolichyl-phosphate (udp-n-acetylglucosamine) n-acetylglucosaminephosphotransferase 1 (glcnac-1-p transferase) protein deficiency
AssigneeModerna Therapeutics, 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 dolichyl-phosphate (UDP-N-acetylglucosamine) N-acetylglucosaminephosphotransferase 1 (GlcNAc-1-P transferase) (SEQ ID NO:
- 9985) 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 is dolichyl-phosphate (UDP-N-acetylglucosamine) N-acetylglucosaminephosphotransferase 1 (GlcNAc-1-P transferase) (SEQ ID NO:
- 9985) 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.