EA201600226A1 - Способы и композиции для рнк-направленного лечения вич-инфекции - Google Patents
Способы и композиции для рнк-направленного лечения вич-инфекцииInfo
- Publication number
- EA201600226A1 EA201600226A1 EA201600226A EA201600226A EA201600226A1 EA 201600226 A1 EA201600226 A1 EA 201600226A1 EA 201600226 A EA201600226 A EA 201600226A EA 201600226 A EA201600226 A EA 201600226A EA 201600226 A1 EA201600226 A1 EA 201600226A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- compositions
- rna
- methods
- hiv infection
- directed treatment
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 208000031886 HIV Infections Diseases 0.000 title 1
- 208000037357 HIV infectious disease Diseases 0.000 title 1
- 208000033519 human immunodeficiency virus infectious disease Diseases 0.000 title 1
- 108020005004 Guide RNA Proteins 0.000 abstract 2
- 108091033409 CRISPR Proteins 0.000 abstract 1
- 238000010354 CRISPR gene editing Methods 0.000 abstract 1
- 108010042407 Endonucleases Proteins 0.000 abstract 1
- 102000004533 Endonucleases Human genes 0.000 abstract 1
- 241000725303 Human immunodeficiency virus Species 0.000 abstract 1
- 206010061598 Immunodeficiency Diseases 0.000 abstract 1
- 208000029462 Immunodeficiency disease Diseases 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 230000007813 immunodeficiency Effects 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 230000009385 viral infection Effects 0.000 abstract 1
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- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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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
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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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- A61K9/00—Medicinal preparations characterised by special physical form
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/18—Antivirals for RNA viruses for HIV
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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
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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 [RNase]; Deoxyribonucleases [DNase]
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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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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16061—Methods of inactivation or attenuation
- C12N2740/16063—Methods of inactivation or attenuation by chemical treatment
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Reproductive Health (AREA)
- Urology & Nephrology (AREA)
- Developmental Biology & Embryology (AREA)
- Gynecology & Obstetrics (AREA)
- Cell Biology (AREA)
- AIDS & HIV (AREA)
Abstract
Настоящее изобретение представляет способы и композиции для лечения инфекции, вызванной вирусом иммунодефицита. Композиции включают последовательности выделенных нуклеиновых кислот, содержащие CRISPR-ассоциированную эндонуклеазу и направляющую РНК, где направляющая РНК комплементарна целевой последовательности в вирусе иммунодефицита человека.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361871626P | 2013-08-29 | 2013-08-29 | |
| US201462018441P | 2014-06-27 | 2014-06-27 | |
| US201462026103P | 2014-07-18 | 2014-07-18 | |
| PCT/US2014/053441 WO2015031775A1 (en) | 2013-08-29 | 2014-08-29 | Methods and compositions for rna-guided treatment of hiv infection |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| EA201600226A1 true EA201600226A1 (ru) | 2016-09-30 |
| EA037850B1 EA037850B1 (ru) | 2021-05-27 |
Family
ID=52587370
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA201600226A EA037850B1 (ru) | 2013-08-29 | 2014-08-29 | Способы и композиции для рнк-направленного лечения вич-инфекции |
Country Status (15)
| Country | Link |
|---|---|
| US (24) | US9925248B2 (ru) |
| EP (2) | EP3038661B1 (ru) |
| JP (3) | JP7118588B2 (ru) |
| KR (2) | KR20160060659A (ru) |
| CN (1) | CN106102781A (ru) |
| AU (2) | AU2014312123A1 (ru) |
| BR (1) | BR112016004091A2 (ru) |
| CA (1) | CA2922428A1 (ru) |
| CL (1) | CL2016000424A1 (ru) |
| EA (1) | EA037850B1 (ru) |
| IL (1) | IL244220B (ru) |
| MX (1) | MX2016002586A (ru) |
| SG (2) | SG10201801658XA (ru) |
| WO (1) | WO2015031775A1 (ru) |
| ZA (1) | ZA201601344B (ru) |
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|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | 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 | ヌクレアーゼ切断特異性の評価および改善 |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| EP4299741A3 (en) | 2012-12-12 | 2024-02-28 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| CN104995302B (zh) | 2013-01-16 | 2021-08-31 | 爱默蕾大学 | Cas9-核酸复合物及其相关用途 |
| US10760064B2 (en) | 2013-03-15 | 2020-09-01 | The General Hospital Corporation | RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci |
| KR102210319B1 (ko) | 2013-03-15 | 2021-02-01 | 더 제너럴 하스피탈 코포레이션 | 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화 |
| WO2014204578A1 (en) | 2013-06-21 | 2014-12-24 | The General Hospital Corporation | Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing |
| RS62263B1 (sr) | 2013-04-16 | 2021-09-30 | Regeneron Pharma | Ciljana modifikacija genoma pacova |
| KR20160056869A (ko) | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
| EP3011031B1 (en) | 2013-06-17 | 2020-09-30 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
| WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| CN105492611A (zh) | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| DK3011029T3 (da) | 2013-06-17 | 2020-03-16 | Broad Inst Inc | Administration, modificering og optimering af tandem-guidesystemer, fremgangsmåder og sammensætninger til sekvensmanipulering |
| 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 |
| CN106102781A (zh) | 2013-08-29 | 2016-11-09 | 英联邦高等教育系统天普大学 | 用于hiv感染的rna指导的治疗的方法和组合物 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| PT3066201T (pt) | 2013-11-07 | 2018-06-04 | Massachusetts Inst Technology | Métodos e composições relacionadas com crispr com arng reguladores |
| RU2725520C2 (ru) | 2013-12-11 | 2020-07-02 | Регенерон Фармасьютикалс, Инк. | Способы и композиции для направленной модификации генома |
| CA2932479A1 (en) | 2013-12-12 | 2015-06-18 | The Rockefeller University | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| WO2015089486A2 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| CN106103705A (zh) | 2013-12-12 | 2016-11-09 | 布罗德研究所有限公司 | 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法 |
| WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| KR20250068794A (ko) | 2013-12-12 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
| RU2016136977A (ru) * | 2014-02-18 | 2018-03-20 | Дьюк Юниверсити | Композиции для инактивации репликации вируса и способы их получения и применения |
| CN106574256A (zh) | 2014-05-30 | 2017-04-19 | 斯坦福大学托管董事会 | 用于潜伏病毒感染的递送治疗的组合物和方法 |
| JP6930834B2 (ja) | 2014-06-16 | 2021-09-01 | ザ・ジョンズ・ホプキンス・ユニバーシティー | H1プロモーターを用いるcrisprガイドrnaの発現のための組成物および方法 |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| KR20170070136A (ko) | 2014-10-15 | 2017-06-21 | 리제너론 파마슈티칼스 인코포레이티드 | 만능성 세포를 생성하거나 유지하는 방법 및 조성물 |
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| WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Protected guide rnas (pgrnas) |
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| MA41382A (fr) * | 2015-03-20 | 2017-11-28 | Univ Temple | Édition génique basée sur le système crispr/endonucléase à induction par tat |
| CN104726449A (zh) * | 2015-03-23 | 2015-06-24 | 国家纳米科学中心 | 一种用于预防和/或治疗HIV的CRISPR-Cas9系统及其制备方法和用途 |
| EP3300507A4 (en) * | 2015-04-02 | 2019-03-13 | Agenovir Corporation | GENERIC ADMINISTRATION AND COMPOSITIONS |
| EP3294343A4 (en) | 2015-05-13 | 2018-09-26 | Seattle Children's Hospital, dba Seattle Children's Research Institute | Enhancing endonuclease based gene editing in primary cells |
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| WO2016196282A1 (en) * | 2015-05-29 | 2016-12-08 | Agenovir Corporation | Compositions and methods for cell targeted hpv treatment |
| WO2016196539A2 (en) * | 2015-06-01 | 2016-12-08 | Temple University - Of The Commonwealth System Of Higher Education | Methods and compositions for rna-guided treatment of hiv infection |
| CN104894071A (zh) * | 2015-06-08 | 2015-09-09 | 东华大学 | 一种对gt1-7细胞进行基因编辑的方法 |
| WO2016205759A1 (en) | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
| KR102840885B1 (ko) | 2015-06-18 | 2025-07-30 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적외 효과를 감소시키는 crispr 효소 돌연변이 |
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| KR102668608B1 (ko) | 2015-08-28 | 2024-05-24 | 더 제너럴 하스피탈 코포레이션 | 조작된 crispr-cas9 뉴클레아제 |
| CN108779447A (zh) * | 2015-09-28 | 2018-11-09 | 天普大学-联邦高等教育系统 | 用于hiv感染的rna引导治疗的方法和组合物 |
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