Treatment of primary ciliary dyskinesia with synthetic messenger rna
AssigneeTranscripTx, Inc.
InventorsDavid J. Lockhart, Brandon Wustman, Mirko Hennig, Daniella Ishimaru
Life of this patent
Estimating the term…
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.
Claims24
A method for treating a subject having primary ciliary dyskinesia, the method comprising administering to said subject in need thereof a polynucleotide comprising a sequence that is at least 70% identical to nucleotides 1-1,000 of SEQ ID NO:
- 15, which polynucleotide includes codons that provide for heterologous expression of a dynein axonemal intermediate chain 1 protein within ciliated cells of said subject.
Dependencies are inferred from the claim text.