BR112022008861A2 - Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 - Google Patents
Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1Info
- Publication number
- BR112022008861A2 BR112022008861A2 BR112022008861A BR112022008861A BR112022008861A2 BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2 BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleic acid
- retinoschisin
- cell
- coding sequence
- integrating
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 5
- 108020004707 nucleic acids Proteins 0.000 title abstract 4
- 150000007523 nucleic acids Chemical class 0.000 title abstract 4
- 102000039446 nucleic acids Human genes 0.000 title abstract 4
- 108091026890 Coding region Proteins 0.000 title abstract 3
- 101710111169 Retinoschisin Proteins 0.000 title abstract 3
- 102100039507 Retinoschisin Human genes 0.000 title abstract 3
- -1 CELL Substances 0.000 title abstract 2
- 238000010276 construction Methods 0.000 title abstract 2
- 150000002632 lipids Chemical class 0.000 title abstract 2
- 239000002105 nanoparticle Substances 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 208000017441 X-linked retinoschisis Diseases 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 238000003780 insertion Methods 0.000 abstract 1
- 230000037431 insertion Effects 0.000 abstract 1
- 230000010354 integration Effects 0.000 abstract 1
- 201000007714 retinoschisis Diseases 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
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- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/7056—Lectin superfamily, e.g. CD23, CD72
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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
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- 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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- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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
- C12N15/87—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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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
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- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
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- 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 [CRISPRs]
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- Proteomics, Peptides & Aminoacids (AREA)
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Abstract
CONSTRUTO DE ÁCIDO NUCLEICO, VETOR, NANOPARTÍCULA LIPÍDICA, CÉLULA, COMPOSIÇÃO PARA USO NA EXPRESSÃO DE RETINOSQUISINA, MÉTODO PARA INTEGRAR UMA SEQUÊNCIA DE CODIFICAÇÃO, E, MÉTODO PARA MODIFICAR UM GENE RS1. Trata se de construtos e composições de ácido nucleico que permitem a inserção e/ou expressão de uma sequência de codificação de retinosquisina. São fornecidos agentes de nuclease direcionados aos loci de RS1. São fornecidos, também, composições e métodos de uso de tais construtos para integração em um locus alvo genômico e/ou expressão em uma célula. São fornecidos, também, métodos de tratamento de retinosquise juvenil ligada ao X que usam os construtos e composições de ácido nucleico.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962932608P | 2019-11-08 | 2019-11-08 | |
PCT/US2020/059568 WO2021092513A1 (en) | 2019-11-08 | 2020-11-07 | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
Publications (1)
Publication Number | Publication Date |
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BR112022008861A2 true BR112022008861A2 (pt) | 2022-08-23 |
Family
ID=73654921
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022008861A BR112022008861A2 (pt) | 2019-11-08 | 2020-11-07 | Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 |
Country Status (11)
Country | Link |
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US (1) | US20230001019A1 (pt) |
EP (1) | EP4054651A1 (pt) |
JP (1) | JP2022553573A (pt) |
KR (1) | KR20220097414A (pt) |
CN (1) | CN114746125A (pt) |
AU (1) | AU2020379046A1 (pt) |
BR (1) | BR112022008861A2 (pt) |
CA (1) | CA3156277A1 (pt) |
IL (1) | IL292605A (pt) |
MX (1) | MX2022005572A (pt) |
WO (1) | WO2021092513A1 (pt) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN113025618B (zh) * | 2019-12-24 | 2024-02-06 | 朗信启昇(苏州)生物制药有限公司 | 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用 |
AU2021216451A1 (en) * | 2020-02-07 | 2022-09-29 | Intellia Therapeutics, Inc. | Compositions and methods for kallikrein ( KLKB1) gene editing |
WO2023081739A1 (en) * | 2021-11-03 | 2023-05-11 | Teamedon International, Inc. | Methods of treating human x-linked retinoschisis using gene therapy |
CN116790559B (zh) * | 2023-07-07 | 2023-11-28 | 山东舜丰生物科技有限公司 | 一种融合HNH结构域的V型Cas酶及其应用 |
Family Cites Families (60)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
US20030104526A1 (en) | 1999-03-24 | 2003-06-05 | Qiang Liu | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
US20050144655A1 (en) | 2000-10-31 | 2005-06-30 | Economides Aris N. | Methods of modifying eukaryotic cells |
AU2884102A (en) | 2000-12-07 | 2002-06-18 | Sangamo Biosciences Inc | Regulation of angiogenesis with zinc finger proteins |
CA2435394C (en) | 2001-01-22 | 2018-01-09 | Sangamo Biosciences, Inc. | Modified zinc finger binding proteins |
AU2002225187A1 (en) | 2001-01-22 | 2002-07-30 | Sangamo Biosciences, Inc. | Zinc finger polypeptides and their use |
AU2003251286B2 (en) | 2002-01-23 | 2007-08-16 | The University Of Utah Research Foundation | Targeted chromosomal mutagenesis using zinc finger nucleases |
US20030232410A1 (en) | 2002-03-21 | 2003-12-18 | Monika Liljedahl | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
CA2497913C (en) | 2002-09-05 | 2014-06-03 | California Institute Of Technology | Use of chimeric nucleases to stimulate gene targeting |
US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
US20080131962A1 (en) | 2006-05-25 | 2008-06-05 | Sangamo Biosciences, Inc. | Engineered cleavage half-domains |
CA2579677A1 (en) | 2004-09-16 | 2006-03-30 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
US7951925B2 (en) | 2006-05-25 | 2011-05-31 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
JP5632610B2 (ja) | 2006-12-14 | 2014-11-26 | ダウ アグロサイエンシィズ エルエルシー | 最適化された非正準ジンクフィンガータンパク質 |
WO2008133938A2 (en) | 2007-04-26 | 2008-11-06 | Sangamo Biosciences, Inc. | Targeted integration into the ppp1r12c locus |
CN102037594A (zh) | 2008-04-11 | 2011-04-27 | Utc电力公司 | 燃料电池和具有歧管贮槽的双极板 |
KR101803737B1 (ko) | 2008-12-04 | 2017-12-01 | 상가모 테라퓨틱스, 인코포레이티드 | 징크 핑거 뉴클레아제를 이용한 랫트의 유전체 편집 |
US20110239315A1 (en) | 2009-01-12 | 2011-09-29 | Ulla Bonas | Modular dna-binding domains and methods of use |
EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
JP5932632B2 (ja) | 2009-03-20 | 2016-06-15 | サンガモ バイオサイエンシーズ, インコーポレイテッド | 改変された亜鉛フィンガータンパク質を使用したcxcr4の修飾 |
US8772008B2 (en) | 2009-05-18 | 2014-07-08 | Sangamo Biosciences, Inc. | Methods and compositions for increasing nuclease activity |
US20120178647A1 (en) | 2009-08-03 | 2012-07-12 | The General Hospital Corporation | Engineering of zinc finger arrays by context-dependent assembly |
US8518392B2 (en) | 2009-08-14 | 2013-08-27 | Regeneron Pharmaceuticals, Inc. | Promoter-regulated differentiation-dependent self-deleting cassette |
CA2779858C (en) | 2009-10-29 | 2019-10-29 | Aris N. Economides | Multifunctional alleles |
WO2011072246A2 (en) | 2009-12-10 | 2011-06-16 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
CN102939377B (zh) | 2010-04-26 | 2016-06-08 | 桑格摩生物科学股份有限公司 | 使用锌指核酸酶对Rosa位点进行基因组编辑 |
AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
KR20220056884A (ko) * | 2011-04-22 | 2022-05-06 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법 |
EP3498833B1 (en) | 2011-09-21 | 2023-08-16 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of transgene expression |
EP2771457B1 (en) | 2011-10-27 | 2017-11-22 | Sangamo Therapeutics, Inc. | Methods and compositions for modification of the hprt locus |
WO2013141680A1 (en) | 2012-03-20 | 2013-09-26 | Vilnius University | RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX |
US9637739B2 (en) | 2012-03-20 | 2017-05-02 | Vilnius University | RNA-directed DNA cleavage by the Cas9-crRNA complex |
PT2800811T (pt) | 2012-05-25 | 2017-08-17 | Univ California | Métodos e composições para modificação de adn alvo dirigida por arn e para modulação dirigida por arn de transcrição |
WO2014033644A2 (en) | 2012-08-28 | 2014-03-06 | Novartis Ag | Methods of nuclease-based genetic engineering |
CN110643600A (zh) | 2012-10-23 | 2020-01-03 | 基因工具股份有限公司 | 用于切割靶dna的系统及其用途 |
ES2769310T3 (es) | 2012-12-06 | 2020-06-25 | Sigma Aldrich Co Llc | Modificación y regulación del genoma basada en CRISPR |
US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
MX2015007550A (es) | 2012-12-12 | 2017-02-02 | Broad Inst Inc | Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas. |
EP3553174A1 (en) | 2012-12-17 | 2019-10-16 | President and Fellows of Harvard College | Rna-guided human genome engineering |
US9873893B2 (en) * | 2013-02-15 | 2018-01-23 | The United States Of America As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating genetically linked diseases of the eye |
US10350306B2 (en) * | 2013-02-15 | 2019-07-16 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating genetically linked diseases of the eye |
RU2690352C2 (ru) | 2013-02-20 | 2019-05-31 | Регенерон Фармасютикалс, Инк. | Генетическая модификация крыс |
JP2016507244A (ja) | 2013-02-27 | 2016-03-10 | ヘルムホルツ・ツェントルム・ミュンヒェン・ドイチェス・フォルシュンクスツェントルム・フューア・ゲズントハイト・ウント・ウムベルト(ゲーエムベーハー)Helmholtz Zentrum MuenchenDeutsches Forschungszentrum fuer Gesundheit und Umwelt (GmbH) | Cas9ヌクレアーゼによる卵母細胞における遺伝子編集 |
CN110540991B (zh) | 2013-03-15 | 2023-10-24 | 通用医疗公司 | 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性 |
WO2014165825A2 (en) | 2013-04-04 | 2014-10-09 | President And Fellows Of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
EP3674408A1 (en) | 2014-06-16 | 2020-07-01 | The Johns Hopkins University | Compositions and methods for the expression of crispr guide rnas |
US20150376587A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
US10342761B2 (en) | 2014-07-16 | 2019-07-09 | Novartis Ag | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
CN113337533A (zh) | 2014-12-23 | 2021-09-03 | 先正达参股股份有限公司 | 用于鉴定和富集包含位点特异性基因组修饰的细胞的方法和组合物 |
WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
WO2017004279A2 (en) | 2015-06-29 | 2017-01-05 | Massachusetts Institute Of Technology | Compositions comprising nucleic acids and methods of using the same |
WO2017136794A1 (en) | 2016-02-03 | 2017-08-10 | Massachusetts Institute Of Technology | Structure-guided chemical modification of guide rna and its applications |
JP7245651B2 (ja) | 2016-03-30 | 2023-03-24 | インテリア セラピューティクス,インコーポレイテッド | Crispr/cas構成成分のための脂質ナノ粒子製剤 |
EA201991369A1 (ru) | 2016-12-08 | 2019-12-30 | Интеллиа Терапьютикс, Инк. | Модифицированные направляющие рнк |
KR20200032693A (ko) | 2017-07-31 | 2020-03-26 | 리제너론 파마슈티칼스 인코포레이티드 | Cas-형질전환 마우스 배아 줄기 세포 및 마우스 및 이것의 용도 |
JP2021500863A (ja) | 2017-09-29 | 2021-01-14 | インテリア セラピューティクス,インコーポレイテッド | ゲノム編集用のポリヌクレオチド、組成物および方法 |
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2020
- 2020-11-07 BR BR112022008861A patent/BR112022008861A2/pt not_active Application Discontinuation
- 2020-11-07 MX MX2022005572A patent/MX2022005572A/es unknown
- 2020-11-07 CA CA3156277A patent/CA3156277A1/en active Pending
- 2020-11-07 AU AU2020379046A patent/AU2020379046A1/en active Pending
- 2020-11-07 EP EP20817149.6A patent/EP4054651A1/en active Pending
- 2020-11-07 WO PCT/US2020/059568 patent/WO2021092513A1/en active Application Filing
- 2020-11-07 KR KR1020227016494A patent/KR20220097414A/ko unknown
- 2020-11-07 CN CN202080077587.XA patent/CN114746125A/zh active Pending
- 2020-11-07 US US17/774,284 patent/US20230001019A1/en active Pending
- 2020-11-07 JP JP2022524995A patent/JP2022553573A/ja active Pending
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WO2021092513A1 (en) | 2021-05-14 |
CN114746125A (zh) | 2022-07-12 |
AU2020379046A1 (en) | 2022-04-21 |
CA3156277A1 (en) | 2021-05-14 |
JP2022553573A (ja) | 2022-12-23 |
US20230001019A1 (en) | 2023-01-05 |
IL292605A (en) | 2022-07-01 |
KR20220097414A (ko) | 2022-07-07 |
MX2022005572A (es) | 2022-06-09 |
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