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 pharmaceutical formulation comprising a polynucleotide that is at least 70% homologous to nucleic acids 1-1,000 of SEQ ID NO:
- 15, which composition is formulated for administration to a subject.
The pharmaceutical formulation of claim 11, wherein said formulation includes a therapeutically effective amount of said polynucleotide.
A pharmaceutical formulation comprising:
- a) a nucleic acid construct that is at least 70% homologous to a dynein axonemal intermediate chain 1 polypeptide, which nucleic acid construct includes codons that provide for heterologous or enhanced expression of the dynein axonemal intermediate chain 1 protein within cells of a subject having or at risk of having primary ciliary dyskinesia;
- and
- b) a cationic lipid, a fusogenic lipid, a cholesterol and a polyethylene glycol (PEG) lipid.
Dependencies are inferred from the claim text.