| abstract |
The present disclosure provides a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a ciliopathy, wherein the polyribonucleotide encodes a functional version of a protein a defect of which is associated with said ciliopathy, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory system. Further, the present disclosure relates to a method for analyzing the effect of a polyribonucleotide on ciliogenesis, wherein said polyribonucleotide encodes a protein involved in and/or required for ciliogenesis. |