Treatment of primary ciliary dyskinesia with synthetic messenger RNA
AssigneeTranscripTx, Inc.
InventorsDavid J. Lockhart, Brandon Wustman, Mirko Hennig, Daniella Ishimaru
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Abstract
Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.
Claims65
A composition comprising a polyribonucleotide comprising a sequence that is at least 70% identical to nucleotides 1-1,000 of SEQ ID NO:
- 15, which composition is formulated for administration to a subject, wherein substantially all uridine residues within said polyribonucleotide are replaced with nucleotide analogues.
A composition comprising a polynucleotide comprising a sequence that is at least 70% identical to nucleotides 1-1,000 of SEQ ID NO:
- 15, which composition is formulated for administration to a subject, wherein said polynucleotide comprises 1-methylpseudouridine.
A composition comprising a polynucleotide comprising a sequence that is at least 70% identical to nucleotides 1-1,000 of SEQ ID NO:
- 15, which composition is formulated for administration to a subject, wherein fewer than 15% of nucleotides within said polynucleotide are nucleotide analogues.
Dependencies are inferred from the claim text.