| abstract |
PROBLEM TO BE SOLVED: To provide a material, preparation, production method, and delivery method of CFTR mRNA for induction of CFTR expression, including induction in the lung of a mammal. The present invention is particularly useful for treating cystic fibrosis. The present invention is based, in part, on the development of formulations of CFTR mRNA and non-spontaneous CFTR mRNA, as well as their method of administration which can induce functional CFTR expression in vivo. Compositions, methods, and uses according to the present invention may provide CFTR expression in the lungs of large mammals, along with a preferred safety profile suitable for the effective treatment of cystic fibrosis. [Selection diagram] None |