Oligonucleotide-containing pharmacological compositions and their use

摘要

The present invention relates to methods and compositions containing oligonucleotides suitable for administration to humans and other mammals.

主权项

1. A composition suitable for administration in a mammal suffering from a pathological disorder or disease comprising two or more modified oligonucleotides, wherein
a.) a first and a second modified oligonucleotide each comprise about seven to seventy-five nucleotides containing seven or more contiguous ribose groups linked by achiral 5′ to 3′ internucleotide phosphate linkages, wherein said ribose groups of said modified oligonucleotides have a 2′ modification, and wherein said 5′ and 3′ ends of said modified oligonucleotides are blocked; b.) said first modified oligonucleotide is complementary to a region of a first gene associated with said pathological disorder or disease and said second modified oligonucleotide is complementary to a region of a second gene associated with said pathological disorder or disease, wherein the sequence of each of said modified oligonucleotides is selected from the group consisting of SEQ ID NO: 1, SEQ ID NO: 6, SEQ ID NO: 10, SEQ ID NO: 12 and SEQ ID NO: 24; and c.) wherein said first and second modified oligonucleotides are present in a total amount of less than about 7 mg.