CN109714954A - 用于组织特异性表达的修饰的u6启动子系统 - Google Patents

用于组织特异性表达的修饰的u6启动子系统 Download PDF

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Publication number
CN109714954A
CN109714954A CN201780033090.6A CN201780033090A CN109714954A CN 109714954 A CN109714954 A CN 109714954A CN 201780033090 A CN201780033090 A CN 201780033090A CN 109714954 A CN109714954 A CN 109714954A
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seq
mirna
nucleic acid
acid molecules
sequence
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Chinese (zh)
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S.Q.哈珀
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Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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CN201780033090.6A 2016-04-02 2017-03-31 用于组织特异性表达的修饰的u6启动子系统 Pending CN109714954A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662317524P 2016-04-02 2016-04-02
US62/317524 2016-04-02
PCT/US2017/025614 WO2017173411A1 (en) 2016-04-02 2017-03-31 Modified u6 promoter system for tissue specific expression

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CN109714954A true CN109714954A (zh) 2019-05-03

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US (3) US11345913B2 (enExample)
EP (2) EP3435755B1 (enExample)
JP (2) JP7253379B2 (enExample)
KR (1) KR102453187B1 (enExample)
CN (1) CN109714954A (enExample)
AU (1) AU2017240236C1 (enExample)
BR (1) BR112018070249A2 (enExample)
CA (1) CA3019832C (enExample)
IL (1) IL262056B2 (enExample)
MA (1) MA44528A (enExample)
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CN108823209B (zh) * 2018-07-18 2021-04-09 华中农业大学 骨骼肌特异性启动子及应用
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
KR20210081324A (ko) 2018-08-02 2021-07-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도
CA3110466A1 (en) 2018-08-22 2020-02-27 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene
AU2019419494A1 (en) * 2018-12-31 2021-07-15 Research Institute At Nationwide Children's Hospital DUX4 RNA silencing using RNA targeting CRISPR-Cas13b
BR112022019889A2 (pt) * 2020-04-02 2023-03-07 Mirecule Inc Inibição direcionada com o uso de oligonucleotídeos modificados geneticamente
UY39417A (es) 2020-09-11 2022-03-31 Arrowhead Pharmaceuticals Inc Agentes de arni para inhibir la expresión de dux4, composiciones de dichos agentes, y métodos de uso
US12275941B2 (en) * 2021-04-15 2025-04-15 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of dynamin-1 variants

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RU2768289C2 (ru) 2022-03-23
US20250019697A1 (en) 2025-01-16
IL262056B1 (en) 2023-06-01
RU2018137677A (ru) 2020-05-12
RU2018137677A3 (enExample) 2020-07-23
MA44528A (fr) 2019-02-06
US20190136235A1 (en) 2019-05-09
AU2017240236A1 (en) 2018-10-18
NZ746893A (en) 2025-05-30
IL262056A (en) 2018-11-29
EP3435755A1 (en) 2019-02-06
KR20180130550A (ko) 2018-12-07
AU2017240236B2 (en) 2023-07-27
EP3435755B1 (en) 2023-11-01
CA3019832A1 (en) 2017-10-05
US11939579B2 (en) 2024-03-26
US11345913B2 (en) 2022-05-31
JP2022046792A (ja) 2022-03-23
CA3019832C (en) 2023-05-09
EP4335502A2 (en) 2024-03-13
JP2019515663A (ja) 2019-06-13
EP3435755A4 (en) 2019-11-27
BR112018070249A2 (pt) 2019-01-29
JP7253379B2 (ja) 2023-04-06
EP4335502A3 (en) 2024-07-03
WO2017173411A1 (en) 2017-10-05
IL262056B2 (en) 2023-10-01
AU2017240236C1 (en) 2024-03-07
US20230077409A1 (en) 2023-03-16
KR102453187B1 (ko) 2022-10-07

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