CN111108201B - 结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸 - Google Patents

结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸 Download PDF

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CN111108201B
CN111108201B CN201880061455.0A CN201880061455A CN111108201B CN 111108201 B CN111108201 B CN 111108201B CN 201880061455 A CN201880061455 A CN 201880061455A CN 111108201 B CN111108201 B CN 111108201B
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antisense oligonucleotide
exon
suitably
dystrophin
seq
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CN111108201A (zh
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横田俊文
越后谷裕介
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University of Alberta
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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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CN201880061455.0A 2017-07-21 2018-07-20 结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸 Active CN111108201B (zh)

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CN202311664929.XA CN117701566A (zh) 2017-07-21 2018-07-20 结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸

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GBGB1711809.2A GB201711809D0 (en) 2017-07-21 2017-07-21 Antisense oligonucleotide
GB1711809.2 2017-07-21
PCT/CA2018/050881 WO2019014772A1 (en) 2017-07-21 2018-07-20 ANTISENSE OLIGONUCLEOTIDES THAT BIND EXON 51 OF HUMAN DYSTROPHIN PRE-MRNA

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CN111108201B true CN111108201B (zh) 2023-12-19

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CN202311664929.XA Pending CN117701566A (zh) 2017-07-21 2018-07-20 结合至人类肌养蛋白前体mRNA的外显子51的反义寡核苷酸

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US (3) US11142767B2 (https=)
EP (1) EP3655535A4 (https=)
JP (3) JP7365336B2 (https=)
KR (1) KR102655989B1 (https=)
CN (2) CN111108201B (https=)
CA (1) CA3105864A1 (https=)
GB (1) GB201711809D0 (https=)
WO (1) WO2019014772A1 (https=)

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MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN113677374A (zh) * 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
WO2021211734A1 (en) * 2020-04-15 2021-10-21 The Regents Of The University Of California Compositions and methods for treatment
AU2021354760A1 (en) * 2020-09-30 2023-05-11 Biomarin Technologies B.V. Antisense oligonucleotides targeting the exon 51 of dystrophin gene
EP3978608A1 (en) * 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
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