JP2022526429A - Amdの処置のためのhtra1調節 - Google Patents
Amdの処置のためのhtra1調節 Download PDFInfo
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- JP2022526429A JP2022526429A JP2021559892A JP2021559892A JP2022526429A JP 2022526429 A JP2022526429 A JP 2022526429A JP 2021559892 A JP2021559892 A JP 2021559892A JP 2021559892 A JP2021559892 A JP 2021559892A JP 2022526429 A JP2022526429 A JP 2022526429A
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- Prior art keywords
- htra1
- sequence
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- protein
- amd
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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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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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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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- 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
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- C—CHEMISTRY; METALLURGY
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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
- C12N15/1137—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 against enzymes
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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
- C12N15/63—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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
- 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
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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
- 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 [RNase]; Deoxyribonucleases [DNase]
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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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
- C12N2310/10—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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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- 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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- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/40—Systems of functionally co-operating vectors
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025093119A JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Amdの処置のためのhtra1調節 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962832182P | 2019-04-10 | 2019-04-10 | |
| US62/832,182 | 2019-04-10 | ||
| PCT/US2020/027802 WO2020210724A1 (en) | 2019-04-10 | 2020-04-10 | Htra1 modulation for treatment of amd |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025093119A Division JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Amdの処置のためのhtra1調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022526429A true JP2022526429A (ja) | 2022-05-24 |
| JPWO2020210724A5 JPWO2020210724A5 (https=) | 2023-04-17 |
| JP2022526429A5 JP2022526429A5 (https=) | 2023-04-17 |
Family
ID=72750902
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021559892A Pending JP2022526429A (ja) | 2019-04-10 | 2020-04-10 | Amdの処置のためのhtra1調節 |
| JP2025093119A Pending JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Amdの処置のためのhtra1調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025093119A Pending JP2025175283A (ja) | 2019-04-10 | 2025-06-04 | Amdの処置のためのhtra1調節 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20230165976A1 (https=) |
| EP (1) | EP3953485A4 (https=) |
| JP (2) | JP2022526429A (https=) |
| CN (1) | CN113966401A (https=) |
| CA (1) | CA3136119A1 (https=) |
| WO (1) | WO2020210724A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113512564A (zh) * | 2021-08-05 | 2021-10-19 | 呈诺再生医学科技(珠海横琴新区)有限公司 | 以iPSC来源的细胞为载体、基因治疗湿性年龄相关性黄斑变性 |
| US20240358855A1 (en) * | 2021-08-18 | 2024-10-31 | University Of Utah Research Foundation | Multigene constructs for treatment of age-related macular degeneration and other complement dysregulation-related conditions |
| US20250003989A1 (en) * | 2021-11-15 | 2025-01-02 | The Children`S Medical Center Corporation | Methods for tauopathy diagnosis and treatment |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009544317A (ja) * | 2006-07-26 | 2009-12-17 | イェール ユニバーシティー | 加齢性黄斑変性の診断および治療 |
| JP2014510526A (ja) * | 2011-03-15 | 2014-05-01 | ユニヴァーシティー オブ ユタ リサーチ ファウンデーション | 血管関連黄斑症およびその症状の診断および治療方法 |
| JP2016536021A (ja) * | 2013-11-07 | 2016-11-24 | エディタス・メディシン,インコーポレイテッド | CRISPR関連方法および支配gRNAのある組成物 |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU8081491A (en) | 1990-06-01 | 1991-12-31 | Cetus Corporation | Compositions and methods for identifying biologically active molecules |
| JP2001524926A (ja) | 1991-09-18 | 2001-12-04 | アフィマックス テクノロジーズ ナームロゼ フェンノートシャップ | オリゴマーの雑多ライブラリーの集合体を合成する方法 |
| US5487994A (en) | 1992-04-03 | 1996-01-30 | The Johns Hopkins University | Insertion and deletion mutants of FokI restriction endonuclease |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
| US5356802A (en) | 1992-04-03 | 1994-10-18 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease |
| ES2204910T3 (es) | 1992-10-01 | 2004-05-01 | The Trustees Of Columbia University In The City Of New York | Bibliotecas quimicas combinatorias complejas codificadas con señales. |
| CN1134156A (zh) | 1993-11-02 | 1996-10-23 | 阿菲马克斯技术公司 | 合成和筛选多种分子 |
| JPH10500112A (ja) | 1994-05-06 | 1998-01-06 | ファーマコピーア,インコーポレイテッド | 組合せ ジヒドロベンゾピラン ライブラリー |
| US5663046A (en) | 1994-06-22 | 1997-09-02 | Pharmacopeia, Inc. | Synthesis of combinatorial libraries |
| DE19710159A1 (de) | 1997-03-12 | 1998-10-01 | Hoechst Ag | In vitro Transkriptionsverfahren zum Screening von Naturstoffen und anderen chemischen Substanzen |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| CA2394850C (en) | 1999-12-06 | 2012-02-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| US20050267061A1 (en) | 2004-04-08 | 2005-12-01 | Sangamo Biosciences, Inc. | Methods and compositions for treating neuropathic and neurodegenerative conditions |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| 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 |
| JP2008506359A (ja) | 2004-04-08 | 2008-03-06 | サンガモ バイオサイエンシズ インコーポレイテッド | ジンクフィンガータンパク質による神経因性疼痛の処置 |
| JP5431151B2 (ja) | 2006-07-13 | 2014-03-05 | ユニバーシティー オブ アイオワ リサーチ ファンデーション | 血管障害および加齢黄斑変性症の治療および診断のための方法および試薬 |
| US7972787B2 (en) | 2007-02-16 | 2011-07-05 | Massachusetts Eye And Ear Infirmary | Methods for detecting age-related macular degeneration |
| TW200911290A (en) * | 2007-07-02 | 2009-03-16 | Alcon Res Ltd | RNAI-mediated inhibition of HTRA1 for treatment of macular degeneration |
| EP2108705A1 (en) * | 2008-04-08 | 2009-10-14 | Universität Duisburg-Essen | Method for analysing the epigenetic status of the HtrA 1 gene in a biological sample |
| CN103917556B (zh) * | 2011-10-14 | 2018-02-06 | 霍夫曼-拉罗奇有限公司 | 抗HtrA1抗体及使用方法 |
| EP2895624B1 (en) | 2012-09-14 | 2022-06-01 | University Of Utah Research Foundation | Methods of predicting the development of amd based on chromosome 1 and chromosome 10 |
| US9546384B2 (en) * | 2013-12-11 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a mouse genome |
| CA2945335A1 (en) | 2014-04-18 | 2015-10-22 | Editas Medicine, Inc. | Crispr-cas-related methods, compositions and components for cancer immunotherapy |
| AU2017374044B2 (en) | 2016-12-08 | 2023-11-30 | Intellia Therapeutics, Inc. | Modified guide RNAs |
| US11674138B2 (en) * | 2017-03-13 | 2023-06-13 | President And Fellows Of Harvard College | Methods of modulating expression of target nucleic acid sequences in a cell |
| EP3824095A4 (en) | 2018-07-20 | 2022-04-20 | University of Utah Research Foundation | GENE THERAPY FOR MACULATE GENERATION |
| US20200291427A1 (en) * | 2018-11-16 | 2020-09-17 | Gemini Therapeutics Inc. | Crispr-based therapeutics for targeting htra1 and methods of use |
| EP4667578A3 (en) * | 2019-04-03 | 2026-04-01 | REGENXBIO Inc. | Gene therapy for eye pathologies |
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2020
- 2020-04-10 WO PCT/US2020/027802 patent/WO2020210724A1/en not_active Ceased
- 2020-04-10 EP EP20787458.7A patent/EP3953485A4/en active Pending
- 2020-04-10 CN CN202080042929.4A patent/CN113966401A/zh active Pending
- 2020-04-10 JP JP2021559892A patent/JP2022526429A/ja active Pending
- 2020-04-10 US US17/599,979 patent/US20230165976A1/en not_active Abandoned
- 2020-04-10 CA CA3136119A patent/CA3136119A1/en active Pending
-
2025
- 2025-06-04 JP JP2025093119A patent/JP2025175283A/ja active Pending
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009544317A (ja) * | 2006-07-26 | 2009-12-17 | イェール ユニバーシティー | 加齢性黄斑変性の診断および治療 |
| JP2014510526A (ja) * | 2011-03-15 | 2014-05-01 | ユニヴァーシティー オブ ユタ リサーチ ファウンデーション | 血管関連黄斑症およびその症状の診断および治療方法 |
| JP2016536021A (ja) * | 2013-11-07 | 2016-11-24 | エディタス・メディシン,インコーポレイテッド | CRISPR関連方法および支配gRNAのある組成物 |
Non-Patent Citations (2)
| Title |
|---|
| LIAO SHA-MEI ET AL.: "Specific correlation between the major chromosome 1 Oq26 haplotype conferring risk for age-related m", MOLECULAR VISION, vol. 23, JPN6024012478, 2017, pages 318 - 333, ISSN: 0005720553 * |
| 第53回日本網膜硝子体学会総会、第31回日本眼循環学会合同大会 プログラム・講演要旨集, JPN6024012477, 2014, pages 89 - 3, ISSN: 0005295731 * |
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| WO2020210724A1 (en) | 2020-10-15 |
| EP3953485A1 (en) | 2022-02-16 |
| US20230165976A1 (en) | 2023-06-01 |
| EP3953485A4 (en) | 2023-05-17 |
| CN113966401A (zh) | 2022-01-21 |
| CA3136119A1 (en) | 2020-10-15 |
| JP2025175283A (ja) | 2025-12-01 |
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