JP2023504190A5 - - Google Patents

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Publication number
JP2023504190A5
JP2023504190A5 JP2022533105A JP2022533105A JP2023504190A5 JP 2023504190 A5 JP2023504190 A5 JP 2023504190A5 JP 2022533105 A JP2022533105 A JP 2022533105A JP 2022533105 A JP2022533105 A JP 2022533105A JP 2023504190 A5 JP2023504190 A5 JP 2023504190A5
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JP
Japan
Prior art keywords
lipid
microparticles
grna molecule
lipid nanoparticles
nanoparticles
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
JP2022533105A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023504190A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/063150 external-priority patent/WO2021113536A1/en
Publication of JP2023504190A publication Critical patent/JP2023504190A/ja
Publication of JP2023504190A5 publication Critical patent/JP2023504190A5/ja
Pending legal-status Critical Current

Links

JP2022533105A 2019-12-03 2020-12-03 遺伝子編集機構の脂質ナノ粒子送達のためのシステムおよび方法 Pending JP2023504190A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962943093P 2019-12-03 2019-12-03
US62/943,093 2019-12-03
PCT/US2020/063150 WO2021113536A1 (en) 2019-12-03 2020-12-03 Systems and methods for lipid nanoparticle delivery of gene editing machinery

Publications (2)

Publication Number Publication Date
JP2023504190A JP2023504190A (ja) 2023-02-01
JP2023504190A5 true JP2023504190A5 (https=) 2023-12-12

Family

ID=76222327

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022533105A Pending JP2023504190A (ja) 2019-12-03 2020-12-03 遺伝子編集機構の脂質ナノ粒子送達のためのシステムおよび方法

Country Status (5)

Country Link
US (1) US20230032846A1 (https=)
EP (1) EP4069314A4 (https=)
JP (1) JP2023504190A (https=)
AR (1) AR122347A1 (https=)
WO (1) WO2021113536A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
KR102787119B1 (ko) 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
AU2016344609B2 (en) * 2015-10-28 2022-05-12 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of duchenne muscular dystrophy
JP7075597B2 (ja) * 2016-05-05 2022-05-26 デューク ユニバーシティ デュシェンヌ型筋ジストロフィーを治療するためのcrispr/cas関連の方法および組成物
EP3707155A2 (en) * 2017-11-09 2020-09-16 Vertex Pharmaceuticals Incorporated Crispr/cas systems for treatment of dmd
AU2019216321A1 (en) * 2018-01-31 2020-07-30 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

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