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ADMINISTRATION D'ARNM AU SNC ET UTILISATIONS ASSOCIEES
专利权人:
INC.;SHIRE HUMAN GENETIC THERAPIES
发明人:
DEROSA, FRANK,HEARTLEIN, MICHAEL,KARVE, SHRIRANG
申请号:
CA2928040
公开号:
CA2928040A1
申请日:
2014.10.22
申请国别(地区):
CA
年份:
2015
代理人:
摘要:
The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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