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
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- 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
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- 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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- 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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- 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
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- 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
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- C12N15/09—Recombinant DNA-technology
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- 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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- 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)
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- Organic Chemistry (AREA)
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- 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 (72)
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US20140031250A1 (en) | 2010-10-07 | 2014-01-30 | David Tsai Ting | Biomarkers of Cancer |
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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 |
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 |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
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WO2015183667A1 (en) * | 2014-05-28 | 2015-12-03 | The Regents Of The University Of California | HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE |
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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 |
EP3256487A4 (en) | 2015-02-09 | 2018-07-18 | Duke University | Compositions and methods for epigenome editing |
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US10626393B2 (en) | 2015-06-04 | 2020-04-21 | Arbutus Biopharma Corporation | 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 |
EP4345454A2 (en) | 2015-08-25 | 2024-04-03 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
US20170088828A1 (en) * | 2015-09-29 | 2017-03-30 | Agenovir Corporation | Compositions and methods for treatment of latent viral infections |
JP2018532403A (ja) * | 2015-09-29 | 2018-11-08 | アジェノビア コーポレーション | 送達方法および組成物 |
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EP3362571A4 (en) | 2015-10-13 | 2019-07-10 | Duke University | GENOMIC ENGINEERING WITH TYPE I CRISPRISMS IN EUKARYOTIC CELLS |
CA3002827A1 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
JP7408284B2 (ja) * | 2015-10-30 | 2024-01-05 | エディタス・メディシン、インコーポレイテッド | 単純ヘルペスウイルスを治療するためのcrispr/cas関連方法及び組成物 |
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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 |
CN110214183A (zh) | 2016-08-03 | 2019-09-06 | 哈佛大学的校长及成员们 | 腺苷核碱基编辑器及其用途 |
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GB2573062A (en) | 2016-10-14 | 2019-10-23 | Harvard College | AAV delivery of nucleobase editors |
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KR102489902B1 (ko) | 2016-11-11 | 2023-01-19 | 바이오 래드 래버러토리스 인코오포레이티드 | 핵산 샘플을 프로세싱하는 방법 |
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US10286084B2 (en) | 2019-05-14 |
PH12016501623A1 (en) | 2016-11-07 |
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