Treatment of amyotrophic lateral sclerosis
AssigneesAssociation Institut De Myologie, Institut National de la Sante et de la Recherche Medicale, Universite Pierre Et Marie Curie (Paris 6)
InventorsMartine Barkats, Maria-Grazia Biferi, Thomas Voit
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Abstract
The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.
Claims34
An antisense oligonucleotide of 20 to 30 nucleotides in length targeting a human SOD1 pre-mRNA, wherein said antisense oligonucleotide comprises SEQ ID NO:1.
A nucleic acid molecule comprising:
- (i) a nucleic acid sequence of SEQ ID NO:1 and (ii) a nucleic acid sequence of SEQ ID NO:4.
An antisense oligonucleotide of 20 to 30 nucleotides in length targeting a human SOD1 pre-mRNA, wherein said antisense oligonucleotide comprises SEQ ID NO:
- 1 and wherein said antisense oligonucleotide is modified with a small nuclear RNA such as the U7 small nuclear RNA.
A vector comprising a nucleic acid that encodes an antisense oligonucleotide of 20 to 30 nucleotides in length targeting a human SOD1 pre-mRNA, wherein said antisense oligonucleotide comprises SEQ ID NO:
- 1.
A vector encoding at least one antisense oligonucleotide targeting a human SOD1 pre-mRNA, wherein said antisense oligonucleotide induces exon-skipping in said pre-mRNA, wherein said vector further comprises an expression cassette containing a nucleotide sequence encoding a human SOD1 protein, wherein said nucleotide sequence comprises SEQ ID NO:11 or SEQ ID NO:12, wherein the antisense oligonucleotide cannot induce exon-skipping in the pre-mRNA encoded by said nucleotide sequence.
A method for treating amyotrophic lateral sclerosis, comprising administering to a subject in need thereof an antisense oligonucleotide of 20 to 30 nucleotides in length targeting a human SOD1 pre-mRNA, wherein said antisense oligonucleotide comprises SEQ ID NO:
- 1.
A nucleic acid sequence comprising the sequence of SEQ ID NO:11 or 12.
A composition comprising an antisense oligonucleotide comprising SEQ ID NO:1 and an antisense oligonucleotide comprising SEQ ID NO:4.
Dependencies are inferred from the claim text.