JP2023500116A - con-希少コドンのためのTREM組成物及び関連する使用 - Google Patents

con-希少コドンのためのTREM組成物及び関連する使用 Download PDF

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JP2023500116A
JP2023500116A JP2022525706A JP2022525706A JP2023500116A JP 2023500116 A JP2023500116 A JP 2023500116A JP 2022525706 A JP2022525706 A JP 2022525706A JP 2022525706 A JP2022525706 A JP 2022525706A JP 2023500116 A JP2023500116 A JP 2023500116A
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trem
con
seq
absent
fragments
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JP2023500116A5 (enExample
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クリスティーン エリザベス ハイディン,
デイビッド アーサー ベリー,
テオニー アナスタシアディス,
ヌーバー ボゴス アフェヤン,
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フラッグシップ パイオニアリング, インコーポレイテッド
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Cosmetics (AREA)
  • Detergent Compositions (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022525706A 2019-11-04 2020-11-04 con-希少コドンのためのTREM組成物及び関連する使用 Pending JP2023500116A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962930361P 2019-11-04 2019-11-04
US62/930,361 2019-11-04
PCT/US2020/058948 WO2021092064A1 (en) 2019-11-04 2020-11-04 Trem compositions for con-rare codons and related uses

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JP2023500116A true JP2023500116A (ja) 2023-01-04
JP2023500116A5 JP2023500116A5 (enExample) 2023-11-14

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US (1) US20220364092A1 (enExample)
EP (1) EP4055163A1 (enExample)
JP (1) JP2023500116A (enExample)
KR (1) KR20220128611A (enExample)
CN (1) CN115003810A (enExample)
AU (1) AU2020379762A1 (enExample)
CA (1) CA3160097A1 (enExample)
MX (1) MX2022005362A (enExample)
WO (1) WO2021092064A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2021243301A2 (en) * 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
KR20230029685A (ko) * 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012006551A2 (en) * 2010-07-08 2012-01-12 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
JP2022502044A (ja) * 2018-09-26 2022-01-11 ケース ウエスタン リザーブ ユニバーシティ タンパク質発現の増加および/またはハプロ不全障害の治療のための方法および組成物
JP2022517275A (ja) * 2019-01-18 2022-03-07 フラッグシップ パイオニアリング, インコーポレイテッド Trem組成物及びその使用

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US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6693086B1 (en) 1998-06-25 2004-02-17 National Jewish Medical And Research Center Systemic immune activation method using nucleic acid-lipid complexes
WO2006105038A2 (en) * 2005-03-25 2006-10-05 The Uab Research Foundation Methods of altering gene expression and methods of treatment utilizing same
GB0600948D0 (en) * 2006-01-18 2006-02-22 Univ Dundee Prevention/Treatment Of Ichthyosis Vulgaris, Atopy And Other Disorders
US20120077224A1 (en) * 2009-06-05 2012-03-29 The Salk Institute For Biological Studies unnatural amino acid incorporation in eukaryotic cells
AU2013327059B2 (en) * 2012-10-02 2018-11-08 Vaxiion Therapeutics, Llc Immunomodulatory minicells and methods of use
CA2930665A1 (en) 2013-11-18 2015-05-21 Rubius Therapeutics, Inc. Synthetic membrane-receiver complexes
MA39819A (fr) 2014-04-01 2017-02-08 Rubius Therapeutics Inc Méthodes et compositions d'immunomodulation
US20180135012A1 (en) 2015-05-13 2018-05-17 Rubius Therapeutics, Inc. Membrane-receiver complex therapeutics
SI3402491T1 (sl) 2016-01-11 2022-05-31 Rubius Therapeutics, Inc. Sestavki in postopki v zvezi z multimodalnimi terapevtskimi celičnimi sistemi za indikacije raka
MX2019000205A (es) 2016-07-07 2019-09-23 Rubius Therapeutics Inc Composiciones y metodos relacionados con sistemas celulares terapeuticos que expresan arn exogeno.
US20180153989A1 (en) 2016-12-02 2018-06-07 Rubius Therapeutics, Inc. Compositions and methods related to cell systems for penetrating solid tumors
CN110520522A (zh) 2017-02-17 2019-11-29 鲁比厄斯治疗法股份有限公司 功能化红系细胞
CA3062426A1 (en) 2017-05-08 2018-11-15 Flagship Pioneering Innovations V, Inc. Compositions for facilitating membrane fusion and uses thereof
WO2020243560A1 (en) * 2019-05-31 2020-12-03 Flagship Pioneering, Inc. Uses of trem compositions to modulate trna pools

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012006551A2 (en) * 2010-07-08 2012-01-12 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
JP2022502044A (ja) * 2018-09-26 2022-01-11 ケース ウエスタン リザーブ ユニバーシティ タンパク質発現の増加および/またはハプロ不全障害の治療のための方法および組成物
JP2022517275A (ja) * 2019-01-18 2022-03-07 フラッグシップ パイオニアリング, インコーポレイテッド Trem組成物及びその使用

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Title
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 478, no. 4, JPN6024044105, 2016, pages 1647 - 1652, ISSN: 0005795510 *
MUTATION RESEARCH, vol. 775, JPN6024044103, 2015, pages 19 - 25, ISSN: 0005451034 *
日本農芸化学会大会講演要旨集, vol. 2017, JPN6024044104, 2017, pages 30 - 02, ISSN: 0005451033 *

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Publication number Publication date
CN115003810A (zh) 2022-09-02
KR20220128611A (ko) 2022-09-21
WO2021092064A1 (en) 2021-05-14
AU2020379762A1 (en) 2022-05-26
MX2022005362A (es) 2022-09-23
US20220364092A1 (en) 2022-11-17
CA3160097A1 (en) 2021-05-14
EP4055163A1 (en) 2022-09-14

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