TREATMENT OF NEURODEGENERATIVE DISEASE THROUGH INTRACRANIAL DELIVERY OF SHORT INTERFERING RNA (SIRNA)

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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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