KR102814622B1 - 단백질 발현 증가용 및/또는 반가불충분성 장애 치료용 방법 및 조성물 - Google Patents

단백질 발현 증가용 및/또는 반가불충분성 장애 치료용 방법 및 조성물 Download PDF

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KR102814622B1
KR102814622B1 KR1020217012452A KR20217012452A KR102814622B1 KR 102814622 B1 KR102814622 B1 KR 102814622B1 KR 1020217012452 A KR1020217012452 A KR 1020217012452A KR 20217012452 A KR20217012452 A KR 20217012452A KR 102814622 B1 KR102814622 B1 KR 102814622B1
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syndrome
trna
gene
certain embodiments
expression
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KR20210091699A (ko
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제프리 엠 콜러
토마스 스위트
하비 로디쉬
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케이스 웨스턴 리저브 유니버시티
화이트헤드 인스티튜트 포 바이오메디칼 리서치
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
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    • 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
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    • 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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  • Neurology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020217012452A 2018-09-26 2019-09-26 단백질 발현 증가용 및/또는 반가불충분성 장애 치료용 방법 및 조성물 Active KR102814622B1 (ko)

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Application Number Priority Date Filing Date Title
US201862736847P 2018-09-26 2018-09-26
US62/736,847 2018-09-26
PCT/US2019/053268 WO2020069199A1 (en) 2018-09-26 2019-09-26 Methods and compositions for increasing protein expression and/or treating a haploinsufficiency disorder

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KR20210091699A KR20210091699A (ko) 2021-07-22
KR102814622B1 true KR102814622B1 (ko) 2025-05-29

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US (2) US12281314B2 (enExample)
EP (1) EP3856905A4 (enExample)
JP (2) JP2022502044A (enExample)
KR (1) KR102814622B1 (enExample)
CN (1) CN113195719A (enExample)
AU (1) AU2019350871B2 (enExample)
BR (1) BR112021005788A2 (enExample)
CA (1) CA3114109A1 (enExample)
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CO (1) CO2021005040A2 (enExample)
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IL (1) IL281848A (enExample)
MX (1) MX2021003567A (enExample)
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ZA (1) ZA202102302B (enExample)

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JP7712270B2 (ja) 2019-11-01 2025-07-23 テバード バイオサイエンシズ インコーポレイテッド 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
CA3160097A1 (en) * 2019-11-04 2021-05-14 Flagship Pioneering, Inc. Trem compositions for con-rare codons and related uses
US20230203510A1 (en) * 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
EP4158032A2 (en) * 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
EP4334450A4 (en) * 2021-05-05 2025-06-18 Tevard Biosciences, Inc. Methods and compositions for treating a premature termination codon-mediated disorder
CA3249261A1 (en) 2022-04-18 2023-10-26 Vertex Pharmaceuticals Incorporated COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER
CN117257958B (zh) * 2023-11-21 2024-02-09 四川大学华西医院 Trps1抑制剂的新用途及用于治疗和/或预防雄激素性秃发的药物
WO2025176166A1 (en) * 2024-02-21 2025-08-28 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna expression cassette
WO2026001270A1 (en) * 2024-06-24 2026-01-02 Zhuhai Codone Biotechnology Co., Ltd. Engineered trna molecules and methods

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JP2006280250A (ja) 2005-03-31 2006-10-19 Toyobo Co Ltd 部位特異的アミノ酸導入法のための新規な直交化ｔＲＮＡ
US20080254540A1 (en) 2007-04-13 2008-10-16 The Salk Institute Ofr Biological Studies METHODS OF GENETICALLY ENCODING UNNATURAL AMINO ACIDS IN EUKARYOTIC CELLS USING ORTHOGONAL tRNA/SYNTHETASE PAIRS

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Also Published As

Publication number Publication date
US20220073933A1 (en) 2022-03-10
KR20210091699A (ko) 2021-07-22
JP2022502044A (ja) 2022-01-11
ZA202102302B (en) 2022-04-28
IL281848A (en) 2021-05-31
AU2019350871A1 (en) 2021-05-20
EP3856905A4 (en) 2023-06-21
CO2021005040A2 (es) 2021-09-20
CA3114109A1 (en) 2020-04-02
AU2019350871B2 (en) 2026-02-26
BR112021005788A2 (pt) 2021-10-26
JP2025010148A (ja) 2025-01-20
US12281314B2 (en) 2025-04-22
CN113195719A (zh) 2021-07-30
WO2020069199A1 (en) 2020-04-02
EA202190736A1 (ru) 2021-09-16
US20260009041A1 (en) 2026-01-08
EP3856905A1 (en) 2021-08-04
CL2021000747A1 (es) 2021-10-22
MX2021003567A (es) 2021-08-24

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