发明名称 ANTISENSE COMPOSITION AND METHOD FOR TREATING MUSCLE ATROPHY
摘要 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 51 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.
申请公布号 WO2006086667(A2) 申请公布日期 2006.08.17
申请号 WO2006US04797 申请日期 2006.02.09
申请人 AVI BIO PHARMA, INC.;IVERSEN, PATRICK, L. 发明人 IVERSEN, PATRICK, L.
分类号 A61K48/00 主分类号 A61K48/00
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