| 摘要 |
PROBLEM TO BE SOLVED: To provide a short chain antisense compound targeting a gene sequence causing a disease.SOLUTION: A short chain antisense compound comprises a compound comprising a high affinity monomer having a monomer length of 8 to 16 and being chemically modified. Such a specific short chain antisense compound with increased efficacy and improved therapeutic index is useful for decreasing target nucleic acid and/or protein in cells, tissues and animals. A short chain antisense compound comprising high affinity nucleotide modification which is useful for decreasing a target RNA in vivo is provided. Such a short chain antisense compound is useful in a dose lower than the antisense compound described before and enables reduction in toxicity and the cost of treatment. Further, the described short chain antisense compound has high efficacy by oral dosing. |
