JP2022501055A - 未成熟終止コドン媒介性障害を治療するための方法および組成物 - Google Patents

未成熟終止コドン媒介性障害を治療するための方法および組成物 Download PDF

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JP2022501055A
JP2022501055A JP2021517046A JP2021517046A JP2022501055A JP 2022501055 A JP2022501055 A JP 2022501055A JP 2021517046 A JP2021517046 A JP 2021517046A JP 2021517046 A JP2021517046 A JP 2021517046A JP 2022501055 A JP2022501055 A JP 2022501055A
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trna
stop codon
expression vector
immature stop
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JP2022501055A5 (https=
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エム． コラー，ジェフリー
スウィート，トーマス
ロディッシュ，ハーベイ
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Whitehead Institute for Biomedical Research
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
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    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
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JP2021517046A 2018-09-26 2019-09-26 未成熟終止コドン媒介性障害を治療するための方法および組成物 Pending JP2022501055A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862736834P 2018-09-26 2018-09-26
US62/736,834 2018-09-26
US201862747646P 2018-10-18 2018-10-18
US62/747,646 2018-10-18
US201962805793P 2019-02-14 2019-02-14
US62/805,793 2019-02-14
PCT/US2019/053260 WO2020069194A1 (en) 2018-09-26 2019-09-26 Methods and compositions for treating a premature stop codon-mediated disorder

Publications (2)

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JP2022501055A true JP2022501055A (ja) 2022-01-06
JP2022501055A5 JP2022501055A5 (https=) 2022-10-04

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JP2021517046A Pending JP2022501055A (ja) 2018-09-26 2019-09-26 未成熟終止コドン媒介性障害を治療するための方法および組成物

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US (1) US20240050594A1 (https=)
EP (1) EP3856253A4 (https=)
JP (1) JP2022501055A (https=)
AU (1) AU2019350869A1 (https=)
CA (1) CA3114108A1 (https=)
WO (1) WO2020069194A1 (https=)

Families Citing this family (7)

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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
MX2022012955A (es) 2020-04-14 2023-02-23 Flagship Pioneering Innovations Vi Llc Composiciones de trem y usos de las mismas.
KR20230029685A (ko) * 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법
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
EP4202045A1 (en) * 2021-12-22 2023-06-28 Universität Hamburg Synthetic transfer rna with modified nucleotides
EP4442826A1 (en) 2023-04-06 2024-10-09 Universität Hamburg Synthetic dna construct encoding transfer rna
EP4458967A1 (en) 2023-05-02 2024-11-06 Universität Hamburg Pharmaceutical composition comprising multiple suppressor transfer rnas

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107177592A (zh) * 2016-03-10 2017-09-19 北京大学 抑制性tRNA通读提前终止密码子疾病中的截短蛋白
US20170354672A1 (en) * 2016-05-16 2017-12-14 The Board Of Regents Of The University Of Texas System Compositions for the delivery of trna as nanoparticles and methods of use therewith
WO2018161032A1 (en) * 2017-03-03 2018-09-07 The Regents Of The University Of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002508959A (ja) * 1998-01-14 2002-03-26 ヒューマン ジーン セラピー リサーチ インスティテュート ヒトのサプレッサーｔＲＮＡオリゴヌクレオチド、及びそれを用いる方法
WO2017049409A1 (en) * 2015-09-25 2017-03-30 The Centre For Drug Research And Development Compositions for promoting readthrough of premature termination codons, and methods of using the same

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107177592A (zh) * 2016-03-10 2017-09-19 北京大学 抑制性tRNA通读提前终止密码子疾病中的截短蛋白
US20170354672A1 (en) * 2016-05-16 2017-12-14 The Board Of Regents Of The University Of Texas System Compositions for the delivery of trna as nanoparticles and methods of use therewith
WO2018161032A1 (en) * 2017-03-03 2018-09-07 The Regents Of The University Of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
WO2019090169A1 (en) * 2017-11-02 2019-05-09 The Wistar Institute Of Anatomy And Biology Methods of rescuing stop codons via genetic reassignment with ace-trna

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US20240050594A1 (en) 2024-02-15
EP3856253A4 (en) 2022-07-06
WO2020069194A1 (en) 2020-04-02
EP3856253A1 (en) 2021-08-04
CA3114108A1 (en) 2020-04-02
AU2019350869A1 (en) 2021-05-20

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