JP7712270B2 - 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 - Google Patents

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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JP7712270B2
JP7712270B2 JP2022526004A JP2022526004A JP7712270B2 JP 7712270 B2 JP7712270 B2 JP 7712270B2 JP 2022526004 A JP2022526004 A JP 2022526004A JP 2022526004 A JP2022526004 A JP 2022526004A JP 7712270 B2 JP7712270 B2 JP 7712270B2
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seq
egfp
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cells
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ピーター エム． エイモン
ショーン マクファーランド
イン－シン チェン
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テバード バイオサイエンシズ インコーポレイテッド
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • 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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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
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  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
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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)

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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)

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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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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

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

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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

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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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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

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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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WO2016149455A2 (en) * 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
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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
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
CN115209902B (zh) 2025-01-07

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