Patent ID: 7498316
Filing Date: 2009-03-03
Classification: A61P,C12N,C12Y

Abstract:
1. A nucleic acid agent comprising, a first polynucleotide sequence encoding an RNAi agent specific for a human SOD1 gain-of function target mRNA, and a second polynucleotide sequence encoding a RNAi resistant replacement gene, wherein the RNAi agent comprises an antisense strand having sufficient sequence complementarity to the gain-of function target mRNA to induce cleavage of the target mRNA, and a sense strand complementary to said antisense strand, wherein the RNAi replacement gene comprises a nucleotide seciuence encoding a wild-type human SOD1, the nucleotide sequence encoding wild-type human SOD1 comprising a sufficient number of silent mutations such that the mRNA encoded by the replacement gene is resistant to cleavage by the RNAi agent, and wherein the antisense strand is encoded by a nucleic acid comprising SEQ ID NO:2 or SEQ ID NO:6.