CN115209902B - 用于治疗提前终止密码子介导的病症的方法和组合物 - Google Patents

用于治疗提前终止密码子介导的病症的方法和组合物 Download PDF

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CN115209902B
CN115209902B CN202080090997.8A CN202080090997A CN115209902B CN 115209902 B CN115209902 B CN 115209902B CN 202080090997 A CN202080090997 A CN 202080090997A CN 115209902 B CN115209902 B CN 115209902B
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trna
seq
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egfp
cells
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CN115209902A (zh
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P.M.埃蒙
S.马克法兰
Y-H.陈
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Twad Bioscience Co ltd
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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CN202080090997.8A 2019-11-01 2020-10-30 用于治疗提前终止密码子介导的病症的方法和组合物 Active CN115209902B (zh)

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US201962929428P 2019-11-01 2019-11-01
US62/929,428 2019-11-01
PCT/US2020/058415 WO2021087401A1 (en) 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder

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CN115209902B true CN115209902B (zh) 2025-01-07

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CN202411915548.9A Pending CN119955780A (zh) 2019-11-01 2020-10-30 用于治疗提前终止密码子介导的病症的方法和组合物

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US (1) US20240148772A1 (https=)
EP (1) EP4051291A4 (https=)
JP (2) JP7712270B2 (https=)
KR (1) KR20220155981A (https=)
CN (2) CN115209902B (https=)
AU (1) AU2020375040A1 (https=)
CA (1) CA3156791A1 (https=)
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US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
MX2022012955A (es) * 2020-04-14 2023-02-23 Flagship Pioneering Innovations Vi Llc Composiciones de trem y usos de las mismas.
AU2022269633A1 (en) * 2021-05-05 2023-11-02 Tevard Biosciences, Inc. Methods and compositions for treating a premature termination codon-mediated disorder
JP2024538097A (ja) * 2021-10-13 2024-10-18 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー Ｔｒｅｍ組成物及び使用方法
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EP4652272A1 (en) * 2023-01-18 2025-11-26 The Broad Institute Inc. Prime editing-mediated readthrough of premature termination codons (pert)
WO2024155745A1 (en) * 2023-01-18 2024-07-25 The Broad Institute, Inc. Base editing-mediated readthrough of premature termination codons (bert)
EP4442826A1 (en) 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
WO2024226381A1 (en) * 2023-04-28 2024-10-31 University Of Rochester A method of treating a premature translation termination codon n1n2n3 (ptc)-related disease by nonsense suppression in a subject
EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas
WO2025111506A1 (en) * 2023-11-22 2025-05-30 Tevard Biosciences, Inc. Modified transfer rnas and methods of use
CN120418432A (zh) * 2023-12-04 2025-08-01 睿愈(北京)生物医药科技有限公司 改善假尿苷修饰的密码子的通读的方法和组合物
WO2026030209A1 (en) * 2024-07-29 2026-02-05 University Of Rochester Use of decoding trnas to boost protein expression or function

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EP4051291A1 (en) 2022-09-07
KR20220155981A (ko) 2022-11-24
WO2021087401A1 (en) 2021-05-06
JP2022554357A (ja) 2022-12-28
CN119955780A (zh) 2025-05-09
AU2020375040A1 (en) 2022-05-19
JP7712270B2 (ja) 2025-07-23
EP4051291A4 (en) 2024-07-03
US20240148772A1 (en) 2024-05-09
CN115209902A (zh) 2022-10-18
JP2025148444A (ja) 2025-10-07
CA3156791A1 (en) 2021-05-06

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