Antisense composition and method for treating muscle atrophy
Abstract
A method and compound for treating skeletal muscle mass deficiency in a human subject are disclosed. The composition is an oligomer of morpholino subunits and phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5′ exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has a base sequence effective to hybridize to an expression-sensitive region of processed or preprocessed human myostatin RNA transcript, identified, in its processed form, by SEQ ID NO:6, and is capable of uptake by target muscle cells in the subject. In practicing the method, the compound is administered in an amount and at a dosage schedule to produce an overall reduction in the level of serum myostatin measured in the patient, and preferably to bring the myostatin level within the a range determined for normal, healthy individuals.
Claims
exact text as granted — not AI-modified1 . (canceled)
2 . An antisense oligomer consisting of a sequence selected from SEQ ID NOS: 1-5.
3 . A pharmaceutical composition comprising the antisense oligomer of claim 2 and a pharmaceutically acceptable carrier.Join the waitlist — get patent alerts
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