OLIGONUCLEOTIDE-CONTAINING PHARMACOLOGICAL COMPOSITIONS AND THEIR USE

摘要

PROBLEM TO BE SOLVED: To provide therapeutic methods and compositions containing oligonucleotides suitable for administration to humans and other mammals.SOLUTION: Provided is a modified oligonucleotide of about 7 to 75 nucleotides including seven or more contiguous ribose groups linked by achiral 5' to 3' internucleoside phosphate linkages, where the modified oligonucleotide complementary to a region of a gene associated with a pathological disorder is selected from the group consisting of the 5' UTR region, the translational start site, the 3' UTR, and the translational termination site. Also provided is a therapeutic method including oral administration of the oligonucleotide.