AU2014312123A1 - Methods and compositions for RNA-guided treatment of HIV infection - Google Patents

Methods and compositions for RNA-guided treatment of HIV infection Download PDF

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Publication number
AU2014312123A1
AU2014312123A1 AU2014312123A AU2014312123A AU2014312123A1 AU 2014312123 A1 AU2014312123 A1 AU 2014312123A1 AU 2014312123 A AU2014312123 A AU 2014312123A AU 2014312123 A AU2014312123 A AU 2014312123A AU 2014312123 A1 AU2014312123 A1 AU 2014312123A1
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Australia
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sequence
ltr
immunodeficiency virus
nucleic acid
human immunodeficiency
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Abandoned
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AU2014312123A
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English (en)
Inventor
Wenhui Hu
Kamel Khalili
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Temple Univ School of Medicine
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Temple Univ School of Medicine
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Abandoned legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/30Special therapeutic applications
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    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16061Methods of inactivation or attenuation
    • C12N2740/16063Methods of inactivation or attenuation by chemical treatment
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)

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  • Health & Medical Sciences (AREA)
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  • Microbiology (AREA)
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  • Immunology (AREA)
  • Physics & Mathematics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Reproductive Health (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Urology & Nephrology (AREA)
  • AIDS & HIV (AREA)
AU2014312123A 2013-08-29 2014-08-29 Methods and compositions for RNA-guided treatment of HIV infection Abandoned AU2014312123A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020264257A AU2020264257B2 (en) 2013-08-29 2020-11-02 Methods and compositions for RNA-guided treatment of HIV infection

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201361871626P 2013-08-29 2013-08-29
US61/871,626 2013-08-29
US201462018441P 2014-06-27 2014-06-27
US62/018,441 2014-06-27
US201462026103P 2014-07-18 2014-07-18
US62/026,103 2014-07-18
PCT/US2014/053441 WO2015031775A1 (en) 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection

Related Child Applications (1)

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AU2020264257A Division AU2020264257B2 (en) 2013-08-29 2020-11-02 Methods and compositions for RNA-guided treatment of HIV infection

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AU2014312123A1 true AU2014312123A1 (en) 2016-03-17

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AU2020264257A Active AU2020264257B2 (en) 2013-08-29 2020-11-02 Methods and compositions for RNA-guided treatment of HIV infection

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US (24) US9925248B2 (enExample)
EP (2) EP4385567A3 (enExample)
JP (3) JP7118588B2 (enExample)
KR (2) KR102829712B1 (enExample)
CN (1) CN106102781A (enExample)
AU (2) AU2014312123A1 (enExample)
BR (1) BR112016004091A2 (enExample)
CA (1) CA2922428A1 (enExample)
CL (1) CL2016000424A1 (enExample)
EA (1) EA037850B1 (enExample)
IL (1) IL244220B (enExample)
MX (1) MX2016002586A (enExample)
SG (2) SG10201801658XA (enExample)
WO (1) WO2015031775A1 (enExample)
ZA (1) ZA201601344B (enExample)

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
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
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
US9885033B2 (en) 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
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
SG10201808935WA (en) 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
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
KR102829712B1 (ko) 2013-08-29 2025-07-10 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
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
CA2930015A1 (en) 2013-11-07 2015-05-14 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
JP6174811B2 (ja) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ゲノムの標的改変のための方法及び組成物
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
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
KR20160089530A (ko) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Hbv 및 바이러스 질병 및 질환을 위한 crispr­cas 시스템 및 조성물의 전달,용도 및 치료적 적용
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
SG11201606819QA (en) * 2014-02-18 2016-09-29 Univ Duke Compositions for the inactivation of virus replication and methods of making and using the same
AU2015266770A1 (en) 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
SG11201610405RA (en) 2014-06-16 2017-01-27 Univ Johns Hopkins Compositions and methods for the expression of crispr guide rnas using the h1 promoter
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3561052A1 (en) 2014-10-15 2019-10-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
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
US20160287678A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
US20180119174A1 (en) 2015-05-13 2018-05-03 Seattle Children's Hospita (dba Seattle Children's Research Institute Enhancing endonuclease based gene editing in primary cells
GB2543873A (en) * 2015-05-29 2017-05-03 Agenovir Corp Compositions and methods for cell targeted HPV treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
WO2016196283A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Antiviral methods and compositions
CA2987927C (en) * 2015-06-01 2024-03-19 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
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的crispr酶突變
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
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
KR102668608B1 (ko) 2015-08-28 2024-05-24 더 제너럴 하스피탈 코포레이션 조작된 crispr-cas9 뉴클레아제
JP2018527943A (ja) * 2015-09-28 2018-09-27 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 Rna誘導性の、hiv感染の処置のための、方法および組成物
WO2017058888A1 (en) * 2015-10-02 2017-04-06 Agenovir Corporation Transgenic nuclease systems and methods
NZ741778A (en) * 2015-10-08 2022-02-25 Harvard College Multiplexed genome editing
AU2016340078A1 (en) * 2015-10-16 2018-04-12 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing Cpf1 for RNA-guided gene editing
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
CA3006305A1 (en) 2015-12-09 2017-06-15 Excision Biotherapeutics, Inc. Gene editing methods and compositions for eliminating risk of jc virus activation and pml (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
EP3407918A4 (en) * 2016-01-25 2019-09-04 Excision Biotherapeutics METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
AU2017219605B2 (en) * 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
SG11201807025SA (en) * 2016-02-26 2018-09-27 Lanzatech New Zealand Ltd Crispr/cas systems for c-1 fixing bacteria
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
CA3024494A1 (en) * 2016-05-10 2017-11-16 United States Government As Represented By The Department Of Veterans Affairs Lentiviral delivery of crispr/cas constructs that cleave genes essential for hiv-1 infection and replication
CN107365786A (zh) * 2016-05-12 2017-11-21 中国科学院微生物研究所 一种将spacer序列克隆至CRISPR-Cas9系统中的方法及其应用
MX2018014172A (es) 2016-05-20 2019-08-22 Regeneron Pharma Métodos para romper la tolerancia inmunológica usando múltiples arn guías.
CA3018294A1 (en) * 2016-06-01 2017-12-07 Excision Biotherapeutics, Inc. Compositions and methods of treatment for lytic and lysogenic viruses
CN109640946A (zh) * 2016-06-03 2019-04-16 天普大学-联邦高等教育系统 通过基因编辑策略进行hiv-1的负反馈调节
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
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
WO2018049372A1 (en) * 2016-09-12 2018-03-15 Excision Biotherapeutics Inc Hiv clinical plan
KR101997116B1 (ko) 2016-10-14 2019-07-05 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
JP7588390B2 (ja) 2016-10-14 2024-11-22 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 核酸塩基エディターのaav送達
WO2018070850A1 (ko) * 2016-10-14 2018-04-19 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US20190071673A1 (en) * 2017-01-18 2019-03-07 Thomas Malcolm CRISPRs WITH IMPROVED SPECIFICITY
WO2018152418A1 (en) * 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
CN106755268B (zh) * 2017-03-22 2020-07-17 中国科学院海洋研究所 体外筛选具有改善记忆力的牡蛎活性肽的方法
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
US20200140865A1 (en) * 2017-04-17 2020-05-07 Temple University - Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
CN111031790A (zh) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 产生基因修改的动物的方法
CN110799525A (zh) 2017-04-21 2020-02-14 通用医疗公司 具有改变的PAM特异性的CPF1(CAS12a)的变体
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP7324713B2 (ja) 2017-05-25 2023-08-10 ザ ジェネラル ホスピタル コーポレイション 改善された精度および特異性を有する塩基エディター
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
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019079819A1 (en) * 2017-10-20 2019-04-25 City Of Hope COMPOSITION AND METHOD FOR ACTIVATION OF LATENT HUMAN IMMUNODEFICIENCY VIRUS (HIV)
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3973056A1 (en) 2019-05-23 2022-03-30 Christiana Care Health Services, Inc. Gene knockout of variant nrf2 for treatment of cancer
WO2020237186A1 (en) 2019-05-23 2020-11-26 Christiana Care Health Services, Inc. Gene knockout of nrf2 for treatment of cancer
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20230257771A1 (en) 2020-04-20 2023-08-17 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023534968A (ja) * 2020-07-13 2023-08-15 アレクサンドラ エル ハウエル 遺伝的に多様なhiv-1単離株に対するcrispr/cas9ガイドrnaの効率及び特異性のための方法及び組成物
WO2022119919A1 (en) * 2020-12-01 2022-06-09 Howell Alexandra Compositions and methods for cleaving viral genomes
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2023091696A1 (en) 2021-11-19 2023-05-25 Christiana Care Gene Editing Institute, Inc. Adenovirus delivery system for cancer treatment
EP4433494A2 (en) 2021-11-19 2024-09-25 Christiana Care Gene Editing Institute, Inc. Methods for intratumoral delivery of crispr/cas systems
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
US20250381213A1 (en) 2022-02-10 2025-12-18 Christiana Care Gene Editing Institute, Inc. Methods for lipid nanoparticle delivery of crispr/cas system
CN114657185B (zh) * 2022-03-28 2023-11-10 福州大学 一种基于适配体有序排列的金磁纳米探针及其在大田软海绵酸检测中的应用
CN115762764A (zh) * 2022-11-25 2023-03-07 中山大学附属第三医院 一种hiv阴性隐球菌脑膜炎治疗结局预测模型及其构建方法
WO2024130151A1 (en) 2022-12-16 2024-06-20 Christiana Care Gene Editing Institute, Inc. Compositions and methods for treating broad chemoresistance through chemoresistance-specific regulatory components
WO2025007140A2 (en) 2023-06-30 2025-01-02 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
US20250319206A1 (en) 2024-04-04 2025-10-16 Christina Care Gene Editing Institute, Inc. Crispr/cas gene editing of neh4 and/or neh5 domains in nrf2
WO2025226816A1 (en) 2024-04-23 2025-10-30 Christiana Care Gene Editing Institute, Inc. Methods of identifying and correlating crispr-induced exon skipping to phenotypic outcomes

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
TW247876B (en) 1993-12-28 1995-05-21 New York Blood Ct Inc Pharmaceutical compositions for prevention or treating HIV-1 or HIV-2 infection
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
WO1997037005A1 (en) * 1996-04-02 1997-10-09 Progenics Pharmaceuticals, Inc. Method for preventing hiv-1 infection of cd4+ cells
US6392029B1 (en) * 1997-05-09 2002-05-21 The Research Foundation Of State University Of New York HIV chemokines
US20040132161A1 (en) * 1998-04-20 2004-07-08 Finkel Terri H. Methods and compositions for increasing CD4lymphocyte immune responsiveness
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
WO2000066759A1 (en) * 1999-04-29 2000-11-09 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6864085B2 (en) 1999-12-14 2005-03-08 Novartis Ag Bovine immunodeficiency virus (BIV) based vectors
FR2825280B1 (fr) 2001-06-01 2005-04-15 Merial Sas Vaccination contre le virus de l'immunodeficience feline
US20030180756A1 (en) 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression
US20070175484A1 (en) 2006-01-30 2007-08-02 Staab Robert J Condoms for beneficial agents delivery
EP1942192A1 (en) 2007-01-08 2008-07-09 Heinrich-Pette-Institut für experimentelle Virologie und Immunologie Use of a tailored recombinase for the treatment of retroviral infections
CN103060378A (zh) 2011-10-24 2013-04-24 四川百利药业有限责任公司 一种siv载体的制备方法
GB201122458D0 (en) * 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
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
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
DK3553174T3 (da) 2012-12-17 2025-08-04 Harvard College Rna-guided modificering af humant genom
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
KR102829712B1 (ko) 2013-08-29 2025-07-10 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
AU2015266770A1 (en) * 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
CA2987927C (en) 2015-06-01 2024-03-19 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
JP2018527943A (ja) 2015-09-28 2018-09-27 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 Rna誘導性の、hiv感染の処置のための、方法および組成物
AU2016340078A1 (en) 2015-10-16 2018-04-12 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing Cpf1 for RNA-guided gene editing
CA3006305A1 (en) 2015-12-09 2017-06-15 Excision Biotherapeutics, Inc. Gene editing methods and compositions for eliminating risk of jc virus activation and pml (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
RU2018130641A (ru) 2016-01-25 2020-02-26 Эксижн Биотерапьютикс Направляемое рнк, удаление вируса jc человека и других полиомавирусов
EP3407918A4 (en) 2016-01-25 2019-09-04 Excision Biotherapeutics METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
AU2017219605B2 (en) 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
WO2017176529A1 (en) 2016-04-06 2017-10-12 Temple Univesity-Of The Commonwealth System Of Higher Education Compositions for eradicating flavivirus infections in subjects
WO2017192172A1 (en) 2016-05-05 2017-11-09 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
EP3466177A4 (en) 2016-05-26 2020-01-29 Nokia Technologies Oy CODEWORD ADJUSTMENT FOR NON-ORIGINAL CODED ACCESS
WO2017213898A2 (en) 2016-06-07 2017-12-14 Temple University - Of The Commonwealth System Of Higher Education Rna guided compositions for preventing and treating hepatitis b virus infections
US20190085326A1 (en) 2016-09-14 2019-03-21 Temple University - Of The Commonwealth System Of Higher Education Negative feedback regulation of HIV-1 by gene editing strategy
US20190365862A1 (en) 2016-10-12 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Combination therapies for eradicating flavivirus infections in subjects
AU2018213952A1 (en) 2017-01-26 2019-08-29 Excision Biotherapeutics, Inc. Lentivirus and non-integrating lentivirus as viral vector to deliver CRISPR therapeutic
WO2018152418A1 (en) 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
US20200140865A1 (en) 2017-04-17 2020-05-07 Temple University - Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
US20210060138A1 (en) 2019-03-06 2021-03-04 Temple University - Of The Commonwealth System Of Higher Education CRISPR and LASER ART Eliminates HIV

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