JP2022526669A - ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 - Google Patents

ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 Download PDF

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JP2022526669A
JP2022526669A JP2021560108A JP2021560108A JP2022526669A JP 2022526669 A JP2022526669 A JP 2022526669A JP 2021560108 A JP2021560108 A JP 2021560108A JP 2021560108 A JP2021560108 A JP 2021560108A JP 2022526669 A JP2022526669 A JP 2022526669A
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cas
crispr
base editing
editing system
based base
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JP2022526669A5 (https=
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チャールズ エイ ガーズバック
ヴェロニカ ゴフ
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Duke University
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
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    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
    • A—HUMAN NECESSITIES
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Reproductive Health (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Neurology (AREA)
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JP2021560108A 2019-04-12 2020-04-12 ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 Pending JP2022526669A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962833454P 2019-04-12 2019-04-12
US62/833,454 2019-04-12
PCT/US2020/027867 WO2020210776A1 (en) 2019-04-12 2020-04-12 Crispr/cas-based base editing composition for restoring dystrophin function

Publications (2)

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JP2022526669A true JP2022526669A (ja) 2022-05-25
JP2022526669A5 JP2022526669A5 (https=) 2023-04-24

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US (1) US20220177879A1 (https=)
EP (1) EP3952884A4 (https=)
JP (1) JP2022526669A (https=)
WO (1) WO2020210776A1 (https=)

Families Citing this family (13)

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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
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
US20240165271A1 (en) * 2021-03-26 2024-05-23 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
CN119912582B (zh) * 2024-06-17 2025-11-04 中国农业大学 单碱基编辑器及其所用脱氨酶与应用

Citations (4)

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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
WO2018098480A1 (en) * 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
WO2018129296A1 (en) * 2017-01-05 2018-07-12 The Board Of Regents Of The University Of Texas System Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences
WO2018179578A1 (ja) * 2017-03-30 2018-10-04 国立大学法人京都大学 ゲノム編集によるエクソンスキッピング誘導方法

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IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
KR102787119B1 (ko) * 2015-11-30 2025-03-27 듀크 유니버시티 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법
WO2018017751A1 (en) * 2016-07-19 2018-01-25 Supereye, Inc. Systems and methods for predictive visual rendering
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WO2018098480A1 (en) * 2016-11-28 2018-05-31 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing
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WO2018179578A1 (ja) * 2017-03-30 2018-10-04 国立大学法人京都大学 ゲノム編集によるエクソンスキッピング誘導方法

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US20220177879A1 (en) 2022-06-09
WO2020210776A1 (en) 2020-10-15
EP3952884A4 (en) 2023-03-22
EP3952884A1 (en) 2022-02-16

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