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公开(公告)号:US20230054720A1
公开(公告)日:2023-02-23
申请号:US17540495
申请日:2021-12-02
发明人: Heidi Rye Hudlebusch , Lykke Pedersen , Erik Funder , Lukasz Kielpinski , Christoffer Sondergaard , Alexander Herbert Stephan , Mads Manso
IPC分类号: A61K31/7088 , C12N15/113 , A61P25/28
摘要: The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia
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公开(公告)号:US20230060373A1
公开(公告)日:2023-03-02
申请号:US17540534
申请日:2021-12-02
发明人: Heidi Rye Hudlebusch , Alexander Herbert Stephan , Lykke Pedersen , Christoffer Sondergaard , Erik Funder
IPC分类号: C12N15/113 , A61P25/28 , A61K31/7115 , A61K31/712 , A61K31/7125
摘要: The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.
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公开(公告)号:US20220177883A1
公开(公告)日:2022-06-09
申请号:US17540569
申请日:2021-12-02
发明人: Heidi Rye Hudlebusch , Lykke Pedersen , Erik Funder , Lukasz Kielpinski , Christoffer Sondergaard , Alexander Herbert Stephan
IPC分类号: C12N15/113 , A61K31/7088 , A61P25/28
摘要: The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.
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