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RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)

  • US 20050277133A1
  • Filed: 02/22/2005
  • Published: 12/15/2005
  • Est. Priority Date: 05/18/2001
  • Status: Abandoned Application
First Claim
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1. A chemically synthesized double stranded short interfering nucleic acid (siNA) molecule that directs cleavage of a huntingtin (HD) RNA via RNA interference, wherein:

  • a. each strand of said RNA molecule is about 19 to about 23 nucleotides in length;

    b. one strand of said RNA molecule comprises nucleotide sequence having sufficient complementarity to said HD RNA for the RNA molecule to direct cleavage of the HD RNA via RNA interference; and

    c. at least one strand of said RNA molecule comprises one or more chemically modified nucleotides.

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