MX2016010781A - Composiciones para la inactivacion de la replicacion de virus y metodos de fabricacion y uso de las mismas. - Google Patents

Composiciones para la inactivacion de la replicacion de virus y metodos de fabricacion y uso de las mismas.

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Publication number
MX2016010781A
MX2016010781A MX2016010781A MX2016010781A MX2016010781A MX 2016010781 A MX2016010781 A MX 2016010781A MX 2016010781 A MX2016010781 A MX 2016010781A MX 2016010781 A MX2016010781 A MX 2016010781A MX 2016010781 A MX2016010781 A MX 2016010781A
Authority
MX
Mexico
Prior art keywords
compositions
methods
target sequence
trna
polynucleotide encoding
Prior art date
Application number
MX2016010781A
Other languages
English (en)
Inventor
F Schinazi Raymond
R Cullen Bryan
Matthew Kennedy E
P Bogerd Hal
Kornepati Anand
Mefferd Adam
Original Assignee
Univ Duke
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Univ Duke filed Critical Univ Duke
Publication of MX2016010781A publication Critical patent/MX2016010781A/es

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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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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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    • C12N15/00—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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    • C12N15/00—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
    • C12N15/1133—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
    • 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)
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  • 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

En el presente documento se proporcionan construcciones recombinantes, vectores y casetes de expresión que incluyen un primer promotor que, convenientemente, es un promotor de ARNt conectado operativamente a un primer polinucleótido que codifica un primer ARN guía único y un segundo promotor conectado operativamente a un segundo polinucleótido que codifica un polipéptido Cas9. El primer ARN guía único incluye una primera parte complementaria a una cadena de una secuencia diana de un virus de ADN y una segunda parte capaz de interaccionar con el polipéptido Cas9. También se proporcionan polipéptidos y polinucleótidos de Cas9 obtenidos de Staphylococcus aureus de codones optimizados con señales de localización nuclear y opcionalmente un marcador de epítopo. También se proporcionan construcciones para la producción de ARNsg que incluyen un ARNt. También se proporcionan métodos para inhibir la replicación viral, inhibir la expresión de una secuencia diana procedente de un virus o tratar una infección vírica o un cáncer inducido por virus usando las composiciones.
MX2016010781A 2014-02-18 2015-02-18 Composiciones para la inactivacion de la replicacion de virus y metodos de fabricacion y uso de las mismas. MX2016010781A (es)

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)

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MX2016010781A true MX2016010781A (es) 2017-02-15

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MX2016010781A MX2016010781A (es) 2014-02-18 2015-02-18 Composiciones para la inactivacion de la replicacion de virus y metodos de fabricacion y uso de las mismas.

Country Status (14)

Country Link
US (2) US10286084B2 (es)
EP (1) EP3107999A4 (es)
JP (1) JP2017506893A (es)
KR (1) KR20160130392A (es)
CN (1) CN106232823A (es)
AU (1) AU2015219167A1 (es)
BR (1) BR112016019068A2 (es)
CA (1) CA2940084A1 (es)
IL (1) IL247309A0 (es)
MX (1) MX2016010781A (es)
PH (1) PH12016501623A1 (es)
RU (1) RU2016136977A (es)
SG (1) SG11201606819QA (es)
WO (1) WO2015126927A2 (es)

Families Citing this family (72)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012048113A2 (en) 2010-10-07 2012-04-12 The General Hospital Corporation Biomarkers of cancer
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
US9163284B2 (en) 2013-08-09 2015-10-20 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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2015183667A1 (en) * 2014-05-28 2015-12-03 The Regents Of The University Of California HYBRID tRNA/pre-miRNA MOLECULES AND METHODS OF USE
WO2015184262A1 (en) 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
WO2016022363A2 (en) 2014-07-30 2016-02-11 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
WO2016196282A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
WO2016197132A1 (en) * 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
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
JP6905755B2 (ja) 2015-08-25 2021-07-21 デューク ユニバーシティ Ｒｎａガイド型エンドヌクレアーゼを使用してゲノム工学における特異性を改善する組成物および方法
CA2999922A1 (en) * 2015-09-29 2017-04-06 Agenovir Corporation Delivery methods and compositions
US20170088828A1 (en) * 2015-09-29 2017-03-30 Agenovir Corporation Compositions and methods for treatment of latent viral infections
JP2018534258A (ja) * 2015-09-29 2018-11-22 アジェノビア コーポレーション 潜伏ウイルスの転写制御のための組成物および方法
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
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
WO2017105991A1 (en) * 2015-12-18 2017-06-22 Danisco Us Inc. Methods and compositions for t-rna based guide rna expression
CA3011270A1 (en) * 2016-01-25 2018-06-14 Temple University Of The Commonwealth System Of Higher Education Rna guided eradication of human jc virus and other polyomaviruses
WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
RU2018144745A (ru) * 2016-06-01 2020-07-09 Эксижн Биотерапьютикс, Инк. Композиции и способы лечения, направленные на литические и лизогенные вирусы
JP2019517503A (ja) * 2016-06-03 2019-06-24 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション 遺伝子編集戦略によるｈｉｖ−１のネガティブフィードバック調節
WO2017223248A1 (en) * 2016-06-21 2017-12-28 University Of Florida Research Foundation, Inc. Method for delivering rna to neurons to treat herpes infections
SG11201900907YA (en) 2016-08-03 2019-02-27 Harvard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
WO2018081504A1 (en) * 2016-10-28 2018-05-03 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus
KR102489902B1 (ko) 2016-11-11 2023-01-19 바이오 래드 래버러토리스 인코오포레이티드 핵산 샘플을 프로세싱하는 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018169983A1 (en) * 2017-03-13 2018-09-20 President And Fellows Of Harvard College Methods of modulating expression of target nucleic acid sequences in a cell
WO2018175502A2 (en) * 2017-03-21 2018-09-27 Shuber Anthony P Treating cancer with cas endonuclease complexes
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
JP2020515258A (ja) * 2017-03-31 2020-05-28 アジェノビア コーポレーション 抗ウイルス治療剤
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2018237369A2 (en) * 2017-06-23 2018-12-27 Vical Incorporated LIPID NANOPARTICLE MEDIA ADMINISTRATION OF PLASMIDIC DNA EXPRESSING CRISPR FOR THE TREATMENT OF CHRONIC INFECTION WITH HEPATITIS B VIRUS
JP2020534795A (ja) 2017-07-28 2020-12-03 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 ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
EP3663310A4 (en) 2017-08-04 2021-08-11 Peking University TALE-RVD WITH SPECIFIC DETECTION OF A DNA BASE MODIFIED BY METHYLATION AND APPLICATION OF IT
US11624077B2 (en) 2017-08-08 2023-04-11 Peking University Gene knockout method
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
CN109468318A (zh) * 2017-09-08 2019-03-15 中山大学 用于抑制HSV-1复制和/或靶标序列表达的CRISPR/Cas9系统、方法、试剂盒及其应用
CN109468319A (zh) * 2017-09-08 2019-03-15 中山大学 用于抑制HSV-1复制和/或靶标序列表达的CRISPR/Cas9系统、方法、试剂盒及其应用
CN107557394A (zh) * 2017-09-29 2018-01-09 南京鼓楼医院 降低CRISPR/Cas9介导的胚胎基因编辑脱靶率的方法
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
KR20200083495A (ko) 2017-10-20 2020-07-08 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
WO2019152868A1 (en) * 2018-02-01 2019-08-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus (hsv) related keratitis
CN111971389A (zh) * 2018-03-27 2020-11-20 苏州克睿基因生物科技有限公司 CRISPR/Cas系统在基因编辑中的应用
WO2019215102A1 (en) * 2018-05-09 2019-11-14 Dsm Ip Assets B.V. Crispr transient expression construct (ctec)
CN111349654B (zh) * 2018-12-20 2023-01-24 北京大学 使用加标签的向导rna构建体进行高效基因筛选的组合物和方法
CN113646434B (zh) * 2018-12-20 2023-05-30 北京大学 使用加标签的向导rna构建体进行高效基因筛选的组合物和方法
WO2020142629A1 (en) * 2019-01-02 2020-07-09 The General Hospital Corporation Reverse transcriptase blocking agents and methods of using the same
WO2020160412A1 (en) * 2019-02-01 2020-08-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions targeting virus genomes
WO2020191153A2 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN114008200A (zh) * 2019-04-18 2022-02-01 株式会社图尔金 用于抑制乙型肝炎病毒增殖的组合物和方法
CN111139240B (zh) * 2020-01-20 2022-03-15 武汉大学 一种靶向乙型肝炎病毒的改造的CRISPR/SaCas9系统及其应用
WO2021178924A1 (en) * 2020-03-05 2021-09-10 Board Of Regents Of The University Of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
KR20230019843A (ko) 2020-05-08 2023-02-09 더 브로드 인스티튜트, 인코퍼레이티드 표적 이중 가닥 뉴클레오티드 서열의 두 가닥의 동시 편집을 위한 방법 및 조성물

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7090976B2 (en) * 1999-11-10 2006-08-15 Rigel Pharmaceuticals, Inc. Methods and compositions comprising Renilla GFP
US20080318210A1 (en) * 2003-08-27 2008-12-25 Rosetta Genomics Bioinformatically detectable group of novel regulatory viral and viral associated oligonucleotides and uses thereof
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
US7795419B2 (en) 2004-05-26 2010-09-14 Rosetta Genomics Ltd. Viral and viral associated miRNAs and uses thereof
JP2008531001A (ja) 2005-02-23 2008-08-14 エージェンシー・フォー・サイエンス・テクノロジー・アンド・リサーチ 不死化フィーダー細胞
WO2008016994A2 (en) 2006-08-01 2008-02-07 Rush University Medical Center Harnessing wnt signaling to restrict hiv replication
WO2009006584A2 (en) * 2007-07-03 2009-01-08 The Regents Of The University Of Colorado Methods and compositions for identifying cellular rna transcripts
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
US9676810B2 (en) * 2010-07-08 2017-06-13 The Brigham And Women's Hospital, Inc. Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules
WO2012139122A1 (en) 2011-04-08 2012-10-11 Azzazy Hassan Mohamed Ei-Said Detection of nucleic acids using unmodified gold nanoparticles
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
PE20150336A1 (es) 2012-05-25 2015-03-25 Univ California Metodos y composiciones para la modificacion de adn objetivo dirigida por arn y para la modulacion de la transcripcion dirigida por arn
CN116064533A (zh) 2012-10-23 2023-05-05 基因工具股份有限公司 用于切割靶dna的组合物及其用途
KR101844123B1 (ko) 2012-12-06 2018-04-02 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
EP2848690B1 (en) 2012-12-12 2020-08-19 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
ES2786193T3 (es) * 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
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.
EP3252160B1 (en) 2012-12-12 2020-10-28 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
ES2553782T3 (es) 2012-12-12 2015-12-11 The Broad Institute, Inc. Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
CN103233028B (zh) * 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
WO2014140347A2 (en) * 2013-03-15 2014-09-18 Allozyne, Inc Novel nucleic acid molecules
KR20160060659A (ko) * 2013-08-29 2016-05-30 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
CN111269902A (zh) * 2013-12-12 2020-06-12 布罗德研究所有限公司 Crispr-cas系统和组合物的递送及用途
BR112017007923B1 (pt) * 2014-10-17 2023-12-12 The Penn State Research Foundation Método para produzir manipulação genética mediada por reações multiplex com rna em uma célula receptora, construção de ácido nucleico,cassete de expressão, vetor, célula receptora e célula geneticamente modificada

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