Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell

摘要

The present invention relates to the field of gene therapy, more specifically to oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell.

主权项

1. A method of making a change in the sequence of a target RNA molecule in a living cell of a human or animal subject, the method comprising providing a single-stranded non-duplexed oligonucleotide capable of making a change in the sequence of the target RNA molecule to a living cell of the subject under conditions that permit uptake of the oligonucleotide by the cell, wherein all nucleosides of the oligonucleotide are 2′-O alkyl ribose ribonucleosides and the oligonucleotide comprises a sequence that is at least partially complementary to the target RNA molecule, to make the change in the target RNA, wherein the change in the sequence of the target RNA comprises an insertion or a substitution of one or more nucleosides, and wherein the target RNA encodes human CFTR protein, and the change in the sequence of the target RNA results in the creation or restoration of a nucleoside triplet coding for phenylalanine at amino acid position 508 of the CFTR protein.