PRODUCTS AND METHODS FOR TREATMENT OF FAMILIAL AMYOTROPHIC LATERAL SCLEROSIS

摘要

The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.

主权项

1. A recombinant adeno-associated virus comprising the superoxide dismutase 1 (SOD1) shRNA-encoding DNA:(SEQ ID NO: 1)GCATCATCAATTTCGAGCAGAAGGAA,(SEQ ID NO: 2)GAAGCATTAAAGGACTGACTGAA,(SEQ ID NO: 3)CTGACTGAAGGCCTGCATGGATT,(SEQ ID NO: 4)CATGGATTCCATGTTCATGA,(SEQ ID NO: 5)GCATGGATTCCATGTTCATGA,(SEQ ID NO: 6)GGTCTGGCCTATAAAGTAGTC,(SEQ ID NO: 7)GGGCATCATCAATTTCGAGCA,(SEQ ID NO: 8)GCATCATCAATTTCGAGCAGA,(SEQ ID NO: 9)GCCTGCATGGATTCCATGTTC,(SEQ ID NO: 10)GGAGGTCTGGCCTATAAAGTA,(SEQ ID NO: 11)GATTCCATGTTCATGAGTTTG,(SEQ ID NO: 12)GGAGATAATACAGCAGGCTGT,(SEQ ID NO: 13)GCTTTAAAGTACCTGTAGTGA,(SEQ ID NO: 14)GCATTAAAGGACTGACTGAAG,(SEQ ID NO: 1)GCATCATCAATTTCGAGCAGAAGGAA,(SEQ ID NO: 2)GAAGCATTAAAGGACTGACTGAA,(SEQ ID NO: 3)CTGACTGAAGGCCTGCATGGATT,(SEQ ID NO: 4)CATGGATTCCATGTTCATGA,(SEQ ID NO: 5)GCATGGATTCCATGTTCATGA,(SEQ ID NO: 6)GGTCTGGCCTATAAAGTAGTC,(SEQ ID NO: 7)GGGCATCATCAATTTCGAGCA,(SEQ ID NO: 8)GCATCATCAATTTCGAGCAGA,(SEQ ID NO: 9)GCCTGCATGGATTCCATGTTC,(SEQ ID NO: 10)GGAGGTCTGGCCTATAAAGTA,(SEQ ID NO: 11)GATTCCATGTTCATGAGTTTG,(SEQ ID NO: 12)GGAGATAATACAGCAGGCTGT,(SEQ ID NO: 13)GCTTTAAAGTACCTGTAGTGA,(SEQ ID NO: 14)GCATTAAAGGACTGACTGAAG,(SEQ ID NO: 15)TCATCAATTTCGAGCAGAA,(SEQ ID NO: 16)TCGAGCAGAAGGAAAGTAA,(SEQ ID NO: 17)GCCTGCATGGATTCCATGT,(SEQ ID NO: 18)TCACTCTCAGGAGACCATT, or(SEQ ID NO: 19)GCTTTAAAGTACCTGTAGT, wherein the recombinant adeno-associated virus genome lacks rep and cap genes.