CN108350454B - 等位基因选择性基因编辑及其用途 - Google Patents
等位基因选择性基因编辑及其用途 Download PDFInfo
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- CN108350454B CN108350454B CN201680067107.5A CN201680067107A CN108350454B CN 108350454 B CN108350454 B CN 108350454B CN 201680067107 A CN201680067107 A CN 201680067107A CN 108350454 B CN108350454 B CN 108350454B
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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
- A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- 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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- A61P19/00—Drugs for skeletal disorders
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- A61P25/00—Drugs for disorders of the nervous system
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
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- Organic Chemistry (AREA)
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- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
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- Cardiology (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562221407P | 2015-09-21 | 2015-09-21 | |
| US62/221,407 | 2015-09-21 | ||
| US201662330827P | 2016-05-02 | 2016-05-02 | |
| US62/330,827 | 2016-05-02 | ||
| PCT/US2016/052889 WO2017053431A2 (en) | 2015-09-21 | 2016-09-21 | Allele selective gene editing and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN108350454A CN108350454A (zh) | 2018-07-31 |
| CN108350454B true CN108350454B (zh) | 2022-05-10 |
Family
ID=58276126
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201680067107.5A Active CN108350454B (zh) | 2015-09-21 | 2016-09-21 | 等位基因选择性基因编辑及其用途 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US10369232B2 (enExample) |
| EP (1) | EP3353298B1 (enExample) |
| JP (1) | JP6799058B2 (enExample) |
| CN (1) | CN108350454B (enExample) |
| ES (1) | ES2959815T3 (enExample) |
| WO (1) | WO2017053431A2 (enExample) |
Families Citing this family (59)
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| EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
| PT3105328T (pt) | 2014-02-11 | 2020-07-06 | Univ Colorado Regents | Engenharia de genomas multiplexada possibilitada por crispr |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CN119320775A (zh) | 2014-12-18 | 2025-01-17 | 综合基因技术公司 | 基于crispr的组合物和使用方法 |
| EP3313989B1 (en) | 2015-06-29 | 2024-12-25 | Ionis Pharmaceuticals, Inc. | Modified crispr rna and modified single crispr rna and uses thereof |
| WO2017053729A1 (en) | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| IL258821B (en) | 2015-10-23 | 2022-07-01 | Harvard College | Nucleobase editors and their uses |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| WO2017223538A1 (en) | 2016-06-24 | 2017-12-28 | The Regents Of The University Of Colorado, A Body Corporate | Methods for generating barcoded combinatorial libraries |
| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| US11242542B2 (en) | 2016-10-07 | 2022-02-08 | Integrated Dna Technologies, Inc. | S. pyogenes Cas9 mutant genes and polypeptides encoded by same |
| KR20230164759A (ko) | 2016-10-07 | 2023-12-04 | 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. | S. 피오게네스 cas9 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드 |
| JP7588390B2 (ja) | 2016-10-14 | 2024-11-22 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 核酸塩基エディターのaav送達 |
| JP2019535287A (ja) | 2016-11-22 | 2019-12-12 | インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド | Crispr/cpf1システム及び方法 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
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| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| CN108977442B (zh) * | 2017-06-05 | 2023-01-06 | 广州市锐博生物科技有限公司 | 用于dna编辑的系统及其应用 |
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| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
| US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
| BR112020006428A2 (pt) * | 2017-09-28 | 2020-09-24 | Toolgen Incorporated | manipulação de genoma artificial para regulação de expressão de genes |
| US11572574B2 (en) | 2017-09-28 | 2023-02-07 | Toolgen Incorporated | Artificial genome manipulation for gene expression regulation |
| CN118530993A (zh) * | 2017-09-29 | 2024-08-23 | 因特利亚治疗公司 | 用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法 |
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| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2025166325A1 (en) * | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | MODIFIED GUIDE RNAs |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101679978A (zh) * | 2007-05-22 | 2010-03-24 | Mdrna有限公司 | 羟甲基取代的rna寡核苷酸和rna复合物 |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
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| CN105188767A (zh) * | 2012-07-25 | 2015-12-23 | 布罗德研究所有限公司 | 可诱导的dna结合蛋白和基因组干扰工具及其应用 |
| ES2536353T3 (es) * | 2012-12-12 | 2015-05-22 | The Broad Institute, Inc. | Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias |
| US9957515B2 (en) * | 2013-03-15 | 2018-05-01 | Cibus Us Llc | Methods and compositions for targeted gene modification |
| HK1223401A1 (zh) * | 2013-05-15 | 2017-07-28 | 桑格摩生物科学股份有限公司 | 用於治疗遗传病状的方法和组合物 |
| CN105683379A (zh) * | 2013-06-17 | 2016-06-15 | 布罗德研究所有限公司 | 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化 |
| CN110003066B (zh) | 2013-11-18 | 2021-09-03 | 阿克丘勒斯治疗公司 | 用于rna递送的可电离的阳离子脂质 |
| JP2017501149A (ja) * | 2013-12-12 | 2017-01-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 粒子送達構成成分を用いた障害及び疾患の標的化のためのcrispr−cas系及び組成物の送達、使用及び治療適用 |
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2016
- 2016-09-21 EP EP16849508.3A patent/EP3353298B1/en active Active
- 2016-09-21 JP JP2018515082A patent/JP6799058B2/ja active Active
- 2016-09-21 CN CN201680067107.5A patent/CN108350454B/zh active Active
- 2016-09-21 US US15/272,107 patent/US10369232B2/en active Active
- 2016-09-21 ES ES16849508T patent/ES2959815T3/es active Active
- 2016-09-21 WO PCT/US2016/052889 patent/WO2017053431A2/en not_active Ceased
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101679978A (zh) * | 2007-05-22 | 2010-03-24 | Mdrna有限公司 | 羟甲基取代的rna寡核苷酸和rna复合物 |
| CN104480112A (zh) * | 2007-05-22 | 2015-04-01 | 阿克丘勒斯治疗公司 | 用于治疗的una寡聚体 |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| WO2015048577A2 (en) * | 2013-09-27 | 2015-04-02 | Editas Medicine, Inc. | Crispr-related methods and compositions |
Non-Patent Citations (2)
| Title |
|---|
| Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells;Ayal Hendel等;《Nat Biotechnol.》;20150629;第33卷(第9期);第985-989页 * |
| Unlocked nucleic acid – an RNA modification with broad potential;Anna Pasternak等;《Org Biomol Chem.》;20110521;第9卷(第10期);第3591-3597页 * |
Also Published As
| Publication number | Publication date |
|---|---|
| ES2959815T3 (es) | 2024-02-28 |
| CA3002647A1 (en) | 2017-03-30 |
| CN108350454A (zh) | 2018-07-31 |
| EP3353298A2 (en) | 2018-08-01 |
| US20170080107A1 (en) | 2017-03-23 |
| US10369232B2 (en) | 2019-08-06 |
| JP2018527013A (ja) | 2018-09-20 |
| EP3353298B1 (en) | 2023-09-13 |
| WO2017053431A3 (en) | 2017-06-01 |
| EP3353298A4 (en) | 2019-09-25 |
| WO2017053431A2 (en) | 2017-03-30 |
| JP6799058B2 (ja) | 2020-12-09 |
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