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.
Claims20
A composition comprising a nucleic acid construct encoding a polypeptide at least 95% identical to a coiled-coil domain containing 39 (CCDC39) polypeptide, wherein said composition is formulated for administration to lung cells of a subject, wherein said composition comprises
- a cationic lipid;
- a fusogenic lipid;
- a cholesterol;
- and
- a polyethylene glycol (PEG) lipid;
- and wherein said nucleic acid construct comprises 1-methylpseudouridine.
Dependencies are inferred from the claim text.