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AssigneeSirna Therapeutics, Inc.
InventorsMarija Tadin-Strapps, Tayeba Khan, Walter Richard Strapps, Laura Sepp-Lorenzino, Vasant Jadhav, Duncan Brown
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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.
A method of modulating in vivo expression of a myostatin gene in a subject comprising introducing to said subject by systemic administration an effective amount of a myostatin siNA conjugate, or a pharmaceutical composition comprising said siNA conjugate, wherein the siNA conjugate comprises an siNA molecule that targets a myostatin gene expressed by said subject linked to a lipophilic moiety, and wherein the siNA conjugate mediates RNA interference.
A method of enhancing muscle mass in a subject comprising reducing myostatin levels in said subject by introducing to said subject by systemic administration an effective amount of a myostatin siNA conjugate, or a pharmaceutical composition comprising said siNA conjugate, wherein the siNA conjugate comprises an siNA molecule that targets a myostatin gene expressed by said subject linked to a lipophilic moiety, and wherein the siNA conjugate mediates RNA interference.
A method of enhancing muscle performance mass in a subject comprising reducing myostatin levels in said subject by introducing to said subject by systemic administration an effective amount of a myostatin siNA conjugate, or a pharmaceutical composition comprising said siNA conjugate, wherein the siNA conjugate comprises an siNA molecule that targets a myostatin gene expressed by said subject linked to a lipophilic moiety, and wherein the siNA conjugate mediates RNA interference.
A method of treating a musculoskeletal disease or disorder, or a disease or disorder that results in conditions in which muscle is adversely affected, in a subject comprising reducing myostatin levels in said subject by introducing to said subject by systemic administration an effective amount of a myostatin siNA conjugate, or a pharmaceutical composition comprising said siNA conjugate, wherein the siNA conjugate comprises an siNA molecule that targets a myostatin gene expressed by said subject linked to a lipophilic moiety, and wherein the siNA conjugate mediates RNA interference.
15-18. (canceled)
A double-stranded short interfering nucleic acid (siNA) molecule that inhibits the expression of myostatin, wherein:
A double-stranded short interfering nucleic acid (siNA) molecule that inhibits the expression of myostatin, wherein:
Dependencies are inferred from the claim text.