MRNA for use in treatment of human genetic diseases
Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.
1. A method of treating a subject deficient in a protein, comprising administering a composition comprising an mRNA and a transfer vehicle, wherein the mRNA encodes a protein corresponding to the protein which is deficient in the subject, and wherein the mRNA comprises a 3′untranslated region (3′-UTR) consisting of SEQ ID NO: 3, and wherein said protein is secreted from said target cell.
2. A method of treating a subject deficient in a protein, comprising administering a composition comprising an mRNA and a transfer vehicle, wherein the mRNA encodes a protein corresponding to the protein which is deficient in the subject, and wherein the mRNA comprises a 3′untranslated region (3′-UTR), consisting of SEQ ID NO: 3, and wherein said mRNA encodes ornithine carbamoyltransferase.
3. The method of claim 1 , wherein the mRNA further comprises a poly A tail.