| 摘要 |
<p>Composition (A) for administering to an animal comprises a modified oligonucleotide (ON), containing 7-75 consecutive ribose groups, linked together by achiral 5' to 3' internucleotide phosphate bonds. ON is complementary to a region (R) of a gene (I) that encodes a protein (II) involved in a muscular atrophy condition, where R is the 5'- or 3'-untranslated region (UTR), or the translation start or termination sites, is new. An independent claim is also included for a kit containing (A) and optionally instructions for use. ACTIVITY : Muscular-Gen.. Female mice were injected with 5 mg/kg of: (a) oligoA, directed against the 5'-untranslated region of the myostatin gene; (b) oligoB, directed against the translation start sequence; (c) oligoC, directed against the translation stop region; or (d) a mixture of all 3. The treatment was intravenous on day 1; intraperitoneal on days 2 and 3, and the treatment was repeated for 10 cycles, but without intravenous doses after the 5th cycle. At the end of the test, the increase in weight of the quadriceps muscle was 0.8% in (a); 2.4% in (b); 7.2% in (c) and 8.8% in (d). MECHANISM OF ACTION : Inhibiting expression of myostatin or other genes involved in muscle wasting conditions.</p> |
