Antisense oligomer treatment for bone loss
Patent Information
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- PERRON INST FOR NEUROLOGICAL & TRANSLATIONAL SCI LTD
- Filing Date
- 2024-06-18
- Publication Date
- 2026-07-06
AI Technical Summary
【0017】 対象における骨疾患の作用を処置する、防止する、または緩和するためのキットであって、その使用のための指示と共に、好適な容器中に詰められた、少なくとも本明細書に記載のアンチセンスオリゴマーおよびその組合せまたはカクテルを含む、キットも提供される。 本発明の開示のさらなる特徴は、以下におけるその数個の非限定的な実施形態の説明でより詳細に記載される。この説明は、単に本発明の開示を例示する目的で含まれるものである。これは、上記で詳述したような本開示の広範な要約、開示または説明への限定として理解されるべきではない。説明は、添付の図面を参照しながらなされる。
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Abstract
Claims
1. Isolated or purified antisense oligomers, as well as combinations and cocktails, for modifying premRNA splicing or mRNA translation in the SOST gene transcript or a portion thereof to induce downregulation of functional SOST protein production.
2. The antisense oligomer according to claim 1 for inducing the production of a shortened protein, the production of a protein lacking a functional region, or a reduction in the total amount of protein produced.
3. The antisense oligomer according to claim 1, which is a phosphorodiamidate morpholino oligomer (PMO) or a 2'-O-methylphosphorothioate oligomer (2'-OMePS).
4. i) The sequences listed in Table 1; or ii) Sequence IDs 1-11 An antisense oligomer according to claim 1, selected from the group including the group.
5. A cDNA copy of the antisense oligomer sequence described in claim 1.
6. A vector containing the antisense oligomer sequence described in claim 1 or the cDNA described in claim 5.
7. A cell containing the antisense oligomer sequence described in claim 1, the cDNA described in claim 5, or the vector described in claim 6.
8. A method for manipulating splice factor binding in target protein gene transcripts, a) Prepare one or more of the antisense oligomers described in claim 1, and bind the oligomer(s) to a target nucleic acid site, A step to induce downregulation of the production of functional SOST protein. Methods that include...
9. A pharmaceutical, prophylactic, or therapeutic composition for treating, preventing, or mitigating the effects of leptin dysregulation in a subject, a) one or more antisense oligomers according to claim 1; and b) One or more pharmaceutically acceptable carriers and / or diluents A composition comprising, which induces downregulation of the production of functional SOST proteins.
10. The method according to claim 8 or the composition according to claim 9, wherein the bone disease is related to bone loss in the subject.
11. The method according to claim 8 or the composition according to claim 9, wherein the bone disease is selected from osteoporosis, postmenopausal osteoporosis, osteogenesis imperfecta, and cancer-related bone loss.
12. A method for treating, preventing, or mitigating the effects of bone disease in a subject, a) Administer an effective amount of one or more antisense oligomers according to claim 1 or a pharmaceutical composition containing one or more antisense oligomers according to claim 1 to the subject, A step to induce downregulation of the production of functional SOST protein. Methods that include...
13. Use of the purified and isolated antisense oligomer according to claim 1 for manufacturing a medicament for treating, preventing, or mitigating the effects of bone disease in a subject.
14. A kit for treating, preventing, or mitigating the effects of bone disease in a subject, comprising, together with instructions for use, an antisense oligomer according to at least claim 1, packaged in a suitable container.