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Antisense composition and method for treating muscle atrophy
专利权人:
Sarepta Therapeutics; Inc.
发明人:
Iversen, Patrick L.,Timmins, Alan P.
申请号:
AU2017236001
公开号:
AU2017236001A1
申请日:
2017.09.29
申请国别(地区):
AU
年份:
2017
代理人:
摘要:
#$%^&*AU2017236001A120171019.pdf#####Il:\szp\Interwoven\NRPortbl\DCC\SZP\15767691_l.docx-29 09/2017 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 51 exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has abase 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 range determined for normal, healthy individuals.
来源网站:
中国工程科技知识中心
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