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TREATMENT OF NEURODEGENERATIVE DISEASE THROUGH INTRACRANIAL DELIVERY OF SHORT INTERFERING RNA (SIRNA)
专利权人:
发明人:
申请号:
EP09013626.8
公开号:
EP2145628B1
申请日:
2003.11.26
申请国别(地区):
EP
年份:
2012
代理人:
摘要:
The present invention provides small interfering RNAs, short hairpin RNAs and vectors and pharmaceutical compositions comprising the siRNA and shRNAs. Methods for treating a neurodegenerative disorder, namely SCA-1 are also described. Particularly, the invention provides a small interfering RNA consisting of a first and second strand wherein said first strand is encoded by a DNA sequence comprising SEQ ID NO. 1 or SEQ ID NO. 2.
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中国工程科技知识中心
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