Antisense nucleic acid and use thereof
AssigneeNiigata University
InventorsAkihiro Sugai, Osamu Onodera
Life of this patent
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Abstract
An antisense nucleic acid targeting intron 6 of TDP-43 mRNA, and including a nucleotide sequence complementary to a sequence consisting of 10 or more consecutive bases in a target sequence, wherein the target sequence is the 96th to 330th or 400th to 530th positions of a nucleotide sequence represented by SEQ ID NO:1.
Claims7
An antisense oligonucleotide targeting intron 6 of TDP-43 pre-mRNA, comprising a nucleotide sequence that is fully complementary to 15 to 25 consecutive bases in a target sequence, wherein
- the target sequence is the 96th to 330th or 400th to 530th positions of a nucleotide sequence represented by SEQ ID NO:1.
A screening method for an alternative splicing enhancer of intron 6 of TDP-43 mRNA, comprising
- culturing CSE1L-knockdown cells expressing TDP-43 mRNA in the presence of a test substance, and
- quantifying splicing variants not including intron 6 and splicing variants including intron 6 among the TDP-43 mRNA in the cells, wherein
- a decrease in expression level of the splicing variants including intron 6 in the presence of the test substance compared to that in the absence of the test substance indicates that the test substance is a candidate for the alternative splicing enhancer of intron 6 of TDP-43 mRNA.
A screening method for a candidate compound for prevention or treatment of TDP-43 proteinopathy, comprising
- culturing CSE1L-knockdown cells expressing TDP-43 mRNA in the presence of a test substance, and
- quantifying splicing variants not including intron 6 and splicing variants including intron 6 among the TDP-43 mRNA in the cells, wherein
- a decrease in expression level of the splicing variants including intron 6 in the presence of the test substance compared to that in the absence of the test substance indicates that the test substance is a candidate compound for prevention or treatment of TDP-43 proteinopathy.
Dependencies are inferred from the claim text.