JP2022554357A5 - - Google Patents

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Publication number
JP2022554357A5
JP2022554357A5 JP2022526004A JP2022526004A JP2022554357A5 JP 2022554357 A5 JP2022554357 A5 JP 2022554357A5 JP 2022526004 A JP2022526004 A JP 2022526004A JP 2022526004 A JP2022526004 A JP 2022526004A JP 2022554357 A5 JP2022554357 A5 JP 2022554357A5
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JP
Japan
Prior art keywords
trna
pharmaceutical composition
nucleotide sequence
expression vector
gene product
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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Application number
JP2022526004A
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English (en)
Japanese (ja)
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JP2022554357A (ja
JP7712270B2 (ja
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Priority claimed from PCT/US2020/058415 external-priority patent/WO2021087401A1/en
Publication of JP2022554357A publication Critical patent/JP2022554357A/ja
Publication of JP2022554357A5 publication Critical patent/JP2022554357A5/ja
Priority to JP2025116258A priority Critical patent/JP2025148444A/ja
Application granted granted Critical
Publication of JP7712270B2 publication Critical patent/JP7712270B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2022526004A 2019-11-01 2020-10-30 未成熟終止コドン媒介障害を処置するための方法および組成物 Active JP7712270B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025116258A JP2025148444A (ja) 2019-11-01 2025-07-10 未成熟終止コドン媒介障害を処置するための方法および組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025116258A Division JP2025148444A (ja) 2019-11-01 2025-07-10 未成熟終止コドン媒介障害を処置するための方法および組成物

Publications (3)

Publication Number Publication Date
JP2022554357A JP2022554357A (ja) 2022-12-28
JP2022554357A5 true JP2022554357A5 (https=) 2024-11-06
JP7712270B2 JP7712270B2 (ja) 2025-07-23

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ID=75715359

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022526004A Active JP7712270B2 (ja) 2019-11-01 2020-10-30 未成熟終止コドン媒介障害を処置するための方法および組成物
JP2025116258A Pending JP2025148444A (ja) 2019-11-01 2025-07-10 未成熟終止コドン媒介障害を処置するための方法および組成物

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JP2025116258A Pending JP2025148444A (ja) 2019-11-01 2025-07-10 未成熟終止コドン媒介障害を処置するための方法および組成物

Country Status (8)

Country Link
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=)
WO (1) WO2021087401A1 (https=)

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Publication number Priority date Publication date Assignee Title
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
KR102930465B1 (ko) 2017-11-02 2026-02-25 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법
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
AU2023255614A1 (en) 2022-04-18 2024-10-24 Vertex Pharmaceuticals Incorporated Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver.
WO2023220342A2 (en) * 2022-05-13 2023-11-16 Shape Therapeutics Inc. Engineered tranfer rnas
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109112126B (zh) * 2010-06-23 2024-06-14 库尔纳公司 通过抑制电压门控钠通道α亚基(SCNA)的天然反义转录物而治疗SCNA相关疾病
MX365525B (es) * 2011-09-06 2019-06-06 Curna Inc Compuestos que regulan la expresión de subunidades alfa de canales de sodio regulados por voltaje en enfermedades relacionadas con epilepsia mioclónica severa de la infancia.
WO2016149455A2 (en) * 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
CN110352244B (zh) * 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
KR102930465B1 (ko) * 2017-11-02 2026-02-25 더 위스타 인스티튜트 오브 아나토미 앤드 바이올로지 ACE-tRNA를 이용한 유전 재할당을 통한 정지 코돈의 구조 방법

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