Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
US10004814B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Nov 10, 2014 |
| Grant date | Jun 26, 2018 |
| Priority date | — |
| Expiry date | Nov 10, 2034 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3515
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.