发明名称 |
METHODS AND COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE |
摘要 |
This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases. |
申请公布号 |
US2015232840(A1) |
申请公布日期 |
2015.08.20 |
申请号 |
US201514589807 |
申请日期 |
2015.01.05 |
申请人 |
UNIVERSITY OF MASSACHUSETTS |
发明人 |
Aronin Neil;Zamore Phillip |
分类号 |
C12N15/113;C12Q1/68;A61K31/7088 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
1. A method of downregulating expression of a target gene in a neural cell distal to the site of administration, the method comprising contacting an iRNA agent with the neural cell for a time sufficient to allow uptake of the iRNA agent into the cell, wherein (i) the iRNA agent comprises a sense and an antisense strand that form an RNA duplex, and (ii) the sequence of the antisense strand of the iRNA agent comprises a nucleotide sequence sufficiently complementary to a target sequence of about 18 to 25 nucleotides of an RNA expressed from the target gene. |
地址 |
Boston MA US |