Methods and Compositions for Treating Neurological Disease

Abstract

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.

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 neural cells for a time sufficient to allow uptake of the iRNA agent into the cells and axonal transport of said iRNA, wherein (i) the iRNA agent comprises a sense and an antisense strand that form an RNA duplex, (ii) the iRNA agent comprises a lipophilic moiety, and (iii) 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. 
     
     
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