JP2023504190A - 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 - Google Patents

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 Download PDF

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JP2023504190A
JP2023504190A JP2022533105A JP2022533105A JP2023504190A JP 2023504190 A JP2023504190 A JP 2023504190A JP 2022533105 A JP2022533105 A JP 2022533105A JP 2022533105 A JP2022533105 A JP 2022533105A JP 2023504190 A JP2023504190 A JP 2023504190A
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lipid
microparticles
lipid nanoparticles
gene
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JP2023504190A5 (https=
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チャールズ エイ ガーズバック
クリストファー ネルソン
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Duke University
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2310/00—Structure or type of the nucleic acid
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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)

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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

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

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US (1) US20230032846A1 (https=)
EP (1) EP4069314A4 (https=)
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AR (1) AR122347A1 (https=)
WO (1) WO2021113536A1 (https=)

Families Citing this family (10)

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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

Citations (3)

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JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
JP2019507579A (ja) * 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
WO2019092505A1 (en) * 2017-11-09 2019-05-16 Casebia Therapeutics Llp Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof

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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
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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JP2016521555A (ja) * 2013-06-05 2016-07-25 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
JP2019507579A (ja) * 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
WO2019092505A1 (en) * 2017-11-09 2019-05-16 Casebia Therapeutics Llp Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof

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US20230032846A1 (en) 2023-02-02
AR122347A1 (es) 2022-09-07
EP4069314A1 (en) 2022-10-12
WO2021113536A1 (en) 2021-06-10
EP4069314A4 (en) 2024-06-26

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