Pharmaceutical Composition for Treating Muscle Disease
AssigneeNational University Corporation Tokyo Medical and Dental University
InventorsTakanori YOKOTA, Tetsuya NAGATA
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Abstract
A nucleic acid complex that exhibits an excellent antisense effect in the skeletal muscle and/or heart muscle, and a composition for treating or preventing a muscle disease that develops in the skeletal muscle, heart muscle, and the like having the nucleic acid complex as an active ingredient is disclosed. Also provided is a double-stranded nucleic acid complex in which a first nucleic acid strand that hybridizes to the transcription product of a target gene and has an antisense effect on the transcription product is annealed with a second nucleic acid strand that has a base sequence complementary to the first nucleic acid strand and is bound to cholesterol or analog thereof.
Claims40
A double-stranded nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein:
- said first nucleic acid strand comprises a base sequence that is capable of hybridizing to all or part of said transcription product of the target gene, and has an antisense effect on said transcription product,
- said second nucleic acid strand comprises a base sequence complementary to said first nucleic acid strand, and is bound to cholesterol or analog thereof, and
- said first nucleic acid strand is annealed to said second nucleic acid strand.
(canceled)
A double-stranded nucleic acid complex comprising a first nucleic acid strand and a second nucleic acid strand, wherein:
- said first nucleic acid strand comprises a base sequence that is capable of hybridizing to all or part of the transcription product of said target gene, and has an antisense effect on said transcription product,
- said second nucleic acid strand comprises a base sequence complementary to said first nucleic acid strand, and
- said first nucleic acid strand is annealed to said second nucleic acid strand.
Dependencies are inferred from the claim text.