JP2022502044A5 - - Google Patents

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Publication number
JP2022502044A5
JP2022502044A5 JP2021517022A JP2021517022A JP2022502044A5 JP 2022502044 A5 JP2022502044 A5 JP 2022502044A5 JP 2021517022 A JP2021517022 A JP 2021517022A JP 2021517022 A JP2021517022 A JP 2021517022A JP 2022502044 A5 JP2022502044 A5 JP 2022502044A5
Authority
JP
Japan
Prior art keywords
syndrome
expression vector
vector according
seq
type
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021517022A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022502044A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/053268 external-priority patent/WO2020069199A1/en
Publication of JP2022502044A publication Critical patent/JP2022502044A/ja
Publication of JP2022502044A5 publication Critical patent/JP2022502044A5/ja
Priority to JP2024170988A priority Critical patent/JP2025010148A/ja
Pending legal-status Critical Current

Links

JP2021517022A 2018-09-26 2019-09-26 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 Pending JP2022502044A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024170988A JP2025010148A (ja) 2018-09-26 2024-09-30 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

Applications Claiming Priority (3)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024170988A Division JP2025010148A (ja) 2018-09-26 2024-09-30 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

Publications (2)

Publication Number Publication Date
JP2022502044A JP2022502044A (ja) 2022-01-11
JP2022502044A5 true JP2022502044A5 (enExample) 2022-10-04

Family

ID=69949913

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021517022A Pending JP2022502044A (ja) 2018-09-26 2019-09-26 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
JP2024170988A Pending JP2025010148A (ja) 2018-09-26 2024-09-30 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

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024170988A Pending JP2025010148A (ja) 2018-09-26 2024-09-30 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

Country Status (15)

Country Link
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)
CL (1) CL2021000747A1 (enExample)
CO (1) CO2021005040A2 (enExample)
EA (1) EA202190736A1 (enExample)
IL (1) IL281848A (enExample)
MX (1) MX2021003567A (enExample)
WO (1) WO2020069199A1 (enExample)
ZA (1) ZA202102302B (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1114149A1 (en) 1998-09-17 2001-07-11 SmithKline Beecham Corporation Rnase p polypeptides
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
JP4406731B2 (ja) * 2005-03-31 2010-02-03 国立大学法人 岡山大学 部位特異的アミノ酸導入法のための新規な直交化tRNA
WO2008127900A1 (en) * 2007-04-13 2008-10-23 The Salk Institute For Biological Studies Methods of genetically encoding unnatural amino acids in eukaryotic cells using orthogonal trna/synthetase pairs
US20120283116A1 (en) * 2009-09-11 2012-11-08 Leppert Mark F Mutant Sodium Channel Nav1.7 and Methods Related Thereto
US9676810B2 (en) * 2010-07-08 2017-06-13 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
US20130129668A1 (en) * 2011-09-01 2013-05-23 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics
WO2014052855A1 (en) 2012-09-27 2014-04-03 Population Diagnostics, Inc. Methods and compositions for screening and treating developmental disorders
EP3052624A1 (en) 2013-10-02 2016-08-10 Wageningen Universiteit Systematic optimization of coding sequence for functional protein expression
EP3362087A1 (en) 2015-10-15 2018-08-22 The Scripps Research Institute BINDING ACTIVITY OF AMINOACYL-tRNA SYNTHETASE IN CHARCOT-MARIE-TOOTH (CMT) NEUROPATHY AND CMT-RELATED NEUROLOGICAL DISEASES
CN116059236A (zh) * 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
WO2017193051A1 (en) 2016-05-06 2017-11-09 The Trustees Of Princeton University Methods of monitoring rnase l activity
PL3458074T3 (pl) * 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
WO2018104385A1 (en) 2016-12-07 2018-06-14 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Codon optimization
JP2022534988A (ja) 2019-05-31 2022-08-04 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 tRNAプールを調節するためのTREMの使用

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