BR112016019068A2 - construto, vetor recombinante, composição farmacêutica, método de inibição de replicação viral ou expressão de uma sequência alvo em uma célula infectada com um vírus, polipeptídeo de sau cas9 recombinante, construto de sau cas9 recombinante, construto recombinante para expressão de um rna guia individual e kit - Google Patents
construto, vetor recombinante, composição farmacêutica, método de inibição de replicação viral ou expressão de uma sequência alvo em uma célula infectada com um vírus, polipeptídeo de sau cas9 recombinante, construto de sau cas9 recombinante, construto recombinante para expressão de um rna guia individual e kitInfo
- Publication number
- BR112016019068A2 BR112016019068A2 BR112016019068A BR112016019068A BR112016019068A2 BR 112016019068 A2 BR112016019068 A2 BR 112016019068A2 BR 112016019068 A BR112016019068 A BR 112016019068A BR 112016019068 A BR112016019068 A BR 112016019068A BR 112016019068 A2 BR112016019068 A2 BR 112016019068A2
- Authority
- BR
- Brazil
- Prior art keywords
- recombinant
- construct
- expression
- target sequence
- individual guide
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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
- C12N15/861—Adenoviral vectors
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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
- 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
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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
- 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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- 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
- A61P31/20—Antivirals for DNA viruses
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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
- A61P31/20—Antivirals for DNA viruses
- A61P31/22—Antivirals for DNA viruses for herpes viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
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- 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
- C12N15/1131—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 viruses
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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
- C12N15/1131—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 viruses
- C12N15/1132—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 viruses against retroviridae, e.g. HIV
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- 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
- C12N15/1131—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 viruses
- C12N15/1133—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 viruses against herpetoviridae, e.g. HSV
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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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- 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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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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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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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- Wood Science & Technology (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Abstract
são fornecidos aqui construtos recombinantes, vetores e cassetes de expressão que incluem um primeiro promotor o qual é, adequadamente, um promotor de trna operativamente ligado a um primeiro polinucleotídeo que codifica um primeiro rna guia individual e um segundo promotor operativamente ligado a um segundo polinucleotídeo que codifica um polipeptídeo de cas9. o primeiro rna guia individual inclui uma primeira porção complementar a uma fita de uma sequência alvo de um vírus de dna e uma segunda porção capaz de interagir com o polipeptídeo de cas9. são fornecidos também polinucleotídeos e polipeptídeos cas9 derivados de staphylococcus aureus com códons otimizados com sinais de localização nuclear e, opcionalmente, um marcador de epítopo. também são fornecidos construtos para produção de sgrnas, incluindo um trna. métodos de inibição de replicação viral, inibição de expressão de uma sequência alvo a partir de um vírus ou tratamento de uma infecção viral ou câncer por meio de indução viral usando as composições também são fornecidos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461940883P | 2014-02-18 | 2014-02-18 | |
PCT/US2015/016354 WO2015126927A2 (en) | 2014-02-18 | 2015-02-18 | Compositions for the inactivation of virus replication and methods of making and using the same |
Publications (1)
Publication Number | Publication Date |
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BR112016019068A2 true BR112016019068A2 (pt) | 2017-10-10 |
Family
ID=53879230
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112016019068A BR112016019068A2 (pt) | 2014-02-18 | 2015-02-18 | construto, vetor recombinante, composição farmacêutica, método de inibição de replicação viral ou expressão de uma sequência alvo em uma célula infectada com um vírus, polipeptídeo de sau cas9 recombinante, construto de sau cas9 recombinante, construto recombinante para expressão de um rna guia individual e kit |
Country Status (14)
Country | Link |
---|---|
US (2) | US10286084B2 (pt) |
EP (1) | EP3107999A4 (pt) |
JP (1) | JP2017506893A (pt) |
KR (1) | KR20160130392A (pt) |
CN (1) | CN106232823A (pt) |
AU (1) | AU2015219167A1 (pt) |
BR (1) | BR112016019068A2 (pt) |
CA (1) | CA2940084A1 (pt) |
IL (1) | IL247309A0 (pt) |
MX (1) | MX2016010781A (pt) |
PH (1) | PH12016501623A1 (pt) |
RU (1) | RU2016136977A (pt) |
SG (1) | SG11201606819QA (pt) |
WO (1) | WO2015126927A2 (pt) |
Families Citing this family (74)
Publication number | Priority date | Publication date | Assignee | Title |
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CN103517990A (zh) | 2010-10-07 | 2014-01-15 | 通用医疗公司 | 癌症生物标志物 |
EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
CN113005148A (zh) | 2013-01-16 | 2021-06-22 | 爱默蕾大学 | Cas9-核酸复合物及其相关用途 |
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 |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
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 |
US9834791B2 (en) | 2013-11-07 | 2017-12-05 | Editas Medicine, Inc. | CRISPR-related methods and compositions with governing gRNAS |
US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
US10619156B2 (en) * | 2014-05-28 | 2020-04-14 | The Regents Of The University Of California | Hybrid tRNA/pre-miRNA molecules and methods of use |
BR112016028023A2 (pt) * | 2014-05-30 | 2017-08-22 | Univ Leland Stanford Junior | Composições e métodos de administração de tratamentos para infecções virais latentes |
AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
MA41349A (fr) * | 2015-01-14 | 2017-11-21 | Univ Temple | Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn |
US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
US10117911B2 (en) * | 2015-05-29 | 2018-11-06 | Agenovir Corporation | Compositions and methods to treat herpes simplex virus infections |
GB2543873A (en) * | 2015-05-29 | 2017-05-03 | Agenovir Corp | Compositions and methods for cell targeted HPV treatment |
US20180245074A1 (en) * | 2015-06-04 | 2018-08-30 | Protiva Biotherapeutics, Inc. | Treating hepatitis b virus infection using crispr |
WO2016197133A1 (en) | 2015-06-04 | 2016-12-08 | Protiva Biotherapeutics, Inc. | Delivering crispr therapeutics with lipid nanoparticles |
EP3325018A4 (en) | 2015-07-22 | 2019-04-24 | Duke University | HIGH EFFICIENCY SCREENING OF REGULATORY ELEMENT FUNCTION USING EPIGENOUS EDITING TECHNOLOGIES |
CA2996001A1 (en) | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
CN108601883A (zh) * | 2015-09-29 | 2018-09-28 | 埃吉诺维亚公司 | 递送方法和组合物 |
AU2016332706A1 (en) * | 2015-09-29 | 2018-04-12 | Agenovir Corporation | Compositions and methods for latent viral transcription regulation |
WO2017058791A1 (en) * | 2015-09-29 | 2017-04-06 | Agenovir Corporation | Compositions and methods for treatment of latent viral infections |
EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
CN108513575A (zh) | 2015-10-23 | 2018-09-07 | 哈佛大学的校长及成员们 | 核碱基编辑器及其用途 |
CN115491373A (zh) * | 2015-10-30 | 2022-12-20 | 爱迪塔斯医药公司 | 治疗单纯疱疹病毒的crispr/cas相关方法及组合物 |
DK3390631T3 (da) * | 2015-12-18 | 2020-07-13 | Danisco Us Inc | Fremgangsmåder og sammensætninger til t-rna-baseret guide-rna-ekspression |
CN108603196A (zh) * | 2016-01-25 | 2018-09-28 | 酶切生物技术公司 | Rna向导的对人类jc病毒和其他多瘤病毒的根除 |
US20190127713A1 (en) * | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR THE TREATMENT OF DISEASES |
CN109069560A (zh) * | 2016-06-01 | 2018-12-21 | 切除生物治疗公司 | 用于裂解性病毒和溶原性病毒的组合物和治疗方法 |
RU2018144877A (ru) * | 2016-06-03 | 2020-07-09 | Темпл Юниверсити - Оф Де Коммонвелт Систем Оф Хаер Эдьюкейшн | Регуляция HIV-1 по механизму отрицательной обратной связи с помощью стратегии редактирования генов |
US11351273B2 (en) | 2016-06-21 | 2022-06-07 | Board Of Supervisors Of Louisiana State University & Agricultural & Mechanical College | Method for delivering RNA to neurons to treat herpes infections |
WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
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 |
KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
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KR20230013167A (ko) | 2016-11-11 | 2023-01-26 | 바이오 래드 래버러토리스 인코오포레이티드 | 핵산 샘플을 프로세싱하는 방법 |
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-
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- 2015-02-18 EP EP15751819.2A patent/EP3107999A4/en not_active Withdrawn
- 2015-02-18 CA CA2940084A patent/CA2940084A1/en not_active Abandoned
- 2015-02-18 SG SG11201606819QA patent/SG11201606819QA/en unknown
- 2015-02-18 JP JP2016552891A patent/JP2017506893A/ja active Pending
- 2015-02-18 US US15/119,867 patent/US10286084B2/en active Active
- 2015-02-18 AU AU2015219167A patent/AU2015219167A1/en not_active Abandoned
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- 2015-02-18 CN CN201580017543.7A patent/CN106232823A/zh active Pending
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RU2016136977A (ru) | 2018-03-20 |
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US10286084B2 (en) | 2019-05-14 |
CN106232823A (zh) | 2016-12-14 |
SG11201606819QA (en) | 2016-09-29 |
EP3107999A2 (en) | 2016-12-28 |
CA2940084A1 (en) | 2015-08-27 |
EP3107999A4 (en) | 2017-10-04 |
JP2017506893A (ja) | 2017-03-16 |
WO2015126927A3 (en) | 2015-11-19 |
MX2016010781A (es) | 2017-02-15 |
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