JP2019520422A5 - - Google Patents

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Publication number
JP2019520422A5
JP2019520422A5 JP2019514195A JP2019514195A JP2019520422A5 JP 2019520422 A5 JP2019520422 A5 JP 2019520422A5 JP 2019514195 A JP2019514195 A JP 2019514195A JP 2019514195 A JP2019514195 A JP 2019514195A JP 2019520422 A5 JP2019520422 A5 JP 2019520422A5
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JP
Japan
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composition
dynein
nucleic acid
acid construct
protein
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JP2019514195A
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English (en)
Japanese (ja)
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JP7672786B2 (ja
JP2019520422A (ja
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Priority claimed from PCT/US2017/034723 external-priority patent/WO2017205767A1/en
Publication of JP2019520422A publication Critical patent/JP2019520422A/ja
Publication of JP2019520422A5 publication Critical patent/JP2019520422A5/ja
Priority to JP2022098210A priority Critical patent/JP2022120180A/ja
Priority to JP2025030716A priority patent/JP2025090635A/ja
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Publication of JP7672786B2 publication Critical patent/JP7672786B2/ja
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JP2019514195A 2016-05-27 2017-05-26 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療 Active JP7672786B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022098210A JP2022120180A (ja) 2016-05-27 2022-06-17 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療
JP2025030716A JP2025090635A (ja) 2016-05-27 2025-02-27 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662342784P 2016-05-27 2016-05-27
US62/342,784 2016-05-27
PCT/US2017/034723 WO2017205767A1 (en) 2016-05-27 2017-05-26 Treatment of primary ciliary dyskinesia with synthetic messenger rna

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022098210A Division JP2022120180A (ja) 2016-05-27 2022-06-17 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療

Publications (3)

Publication Number Publication Date
JP2019520422A JP2019520422A (ja) 2019-07-18
JP2019520422A5 true JP2019520422A5 (https=) 2020-07-02
JP7672786B2 JP7672786B2 (ja) 2025-05-08

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ID=60411943

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019514195A Active JP7672786B2 (ja) 2016-05-27 2017-05-26 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療
JP2022098210A Pending JP2022120180A (ja) 2016-05-27 2022-06-17 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療
JP2025030716A Pending JP2025090635A (ja) 2016-05-27 2025-02-27 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022098210A Pending JP2022120180A (ja) 2016-05-27 2022-06-17 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療
JP2025030716A Pending JP2025090635A (ja) 2016-05-27 2025-02-27 合成メッセンジャーrnaによる原発性繊毛機能不全症の治療

Country Status (10)

Country Link
US (6) US20190117796A1 (https=)
EP (2) EP3463483B1 (https=)
JP (3) JP7672786B2 (https=)
CN (2) CN117018228A (https=)
AU (3) AU2017271665B2 (https=)
CA (1) CA3025626A1 (https=)
DK (1) DK3463483T3 (https=)
ES (1) ES2972405T3 (https=)
PT (1) PT3463483T (https=)
WO (1) WO2017205767A1 (https=)

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CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
KR102832035B1 (ko) * 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
CA3100946A1 (en) 2018-05-22 2019-11-28 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
KR102116449B1 (ko) * 2018-10-01 2020-05-29 동국대학교 산학협력단 Ofd1을 이용한 피부 탈색 질환 치료용 조성물의 스크리닝 방법
JP2022516356A (ja) * 2019-01-07 2022-02-25 トランスレイト バイオ, インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
HUE065017T2 (hu) * 2019-02-14 2024-04-28 Ethris Gmbh Poliribonukleotid ciliogenezisre gyakorolt hatásának elemzése
JP7686573B6 (ja) 2019-05-24 2025-07-04 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド アルツハイマー病のための遺伝子治療
CN110438220A (zh) * 2019-08-16 2019-11-12 深圳市人民医院 纤毛不动综合症基因面板试剂盒及其应用
BR112022010373A2 (pt) * 2019-11-29 2022-08-16 Paros Bio Inc Terapia genética para doenças neurodegenerativas
CN111500697A (zh) * 2020-03-20 2020-08-07 四川省人民医院 不动纤毛综合症筛查试剂盒
EP4146680A1 (en) * 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス,インク. 細胞への標的送達のための組成物および方法
WO2022204215A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
JP2024527541A (ja) * 2021-07-01 2024-07-25 トランスレイト バイオ, インコーポレイテッド Mrnaの送達のための組成物
EP4379055A4 (en) * 2021-07-27 2025-08-06 Sk Bioscience Co Ltd MRNA FOR PROTEIN EXPRESSION AND TEMPLATE FOR IT
WO2023086893A1 (en) * 2021-11-10 2023-05-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
CN115287326B (zh) * 2022-08-05 2025-05-16 中国科学院深圳先进技术研究院 一种评估流感嗜血杆菌感染性的方法
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN119709879B (zh) * 2024-12-27 2025-09-26 天津大学 efcab2基因在构建脑积水动物模型中的应用及模型的构建方法

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WO2006138380A2 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
CN101104853A (zh) * 2007-06-21 2008-01-16 上海交通大学 小鼠Dnaic2基因序列
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WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
WO2019161459A1 (en) 2018-02-26 2019-08-29 Children's Medical Research Institute Methods for codon optimization
WO2020051223A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3846857A4 (en) 2018-09-04 2022-10-12 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
JP2022516356A (ja) 2019-01-07 2022-02-25 トランスレイト バイオ, インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
HUE065017T2 (hu) 2019-02-14 2024-04-28 Ethris Gmbh Poliribonukleotid ciliogenezisre gyakorolt hatásának elemzése
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204215A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof

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