CL2016000424A1 - Métodos y composiciones para el arn-guiado tratamiento de la infección por vih - Google Patents

Métodos y composiciones para el arn-guiado tratamiento de la infección por vih

Info

Publication number
CL2016000424A1
CL2016000424A1 CL2016000424A CL2016000424A CL2016000424A1 CL 2016000424 A1 CL2016000424 A1 CL 2016000424A1 CL 2016000424 A CL2016000424 A CL 2016000424A CL 2016000424 A CL2016000424 A CL 2016000424A CL 2016000424 A1 CL2016000424 A1 CL 2016000424A1
Authority
CL
Chile
Prior art keywords
arn
compositions
methods
hiv infection
guided treatment
Prior art date
Application number
CL2016000424A
Other languages
English (en)
Inventor
Kamel Khalili
Wenhui Hu
Original Assignee
Univ Temple
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.)
Filing date
Publication date
Application filed by Univ Temple filed Critical Univ Temple
Publication of CL2016000424A1 publication Critical patent/CL2016000424A1/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0034—Urogenital system, e.g. vagina, uterus, cervix, penis, scrotum, urethra, bladder; Personal lubricants
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16061—Methods of inactivation or attenuation
    • C12N2740/16063—Methods of inactivation or attenuation by chemical treatment
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Reproductive Health (AREA)
  • Urology & Nephrology (AREA)
  • AIDS & HIV (AREA)
CL2016000424A 2013-08-29 2016-02-25 Métodos y composiciones para el arn-guiado tratamiento de la infección por vih CL2016000424A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361871626P 2013-08-29 2013-08-29
US201462018441P 2014-06-27 2014-06-27
US201462026103P 2014-07-18 2014-07-18

Publications (1)

Publication Number Publication Date
CL2016000424A1 true CL2016000424A1 (es) 2017-07-14

Family

ID=52587370

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016000424A CL2016000424A1 (es) 2013-08-29 2016-02-25 Métodos y composiciones para el arn-guiado tratamiento de la infección por vih

Country Status (15)

Country Link
US (24) US9925248B2 (es)
EP (2) EP3038661B1 (es)
JP (3) JP7118588B2 (es)
KR (2) KR102829712B1 (es)
CN (1) CN106102781A (es)
AU (2) AU2014312123A1 (es)
BR (1) BR112016004091A2 (es)
CA (1) CA2922428A1 (es)
CL (1) CL2016000424A1 (es)
EA (1) EA037850B1 (es)
IL (1) IL244220B (es)
MX (1) MX2016002586A (es)
SG (2) SG11201601313TA (es)
WO (1) WO2015031775A1 (es)
ZA (1) ZA201601344B (es)

Families Citing this family (119)

* 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
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
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
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
AU2014207618A1 (en) 2013-01-16 2015-08-06 Emory University Cas9-nucleic acid complexes and uses related thereto
IL289396B2 (en) 2013-03-15 2023-12-01 The General Hospital Coporation Using truncated guide rnas (tru-grnas) to increase 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
CA2908697C (en) 2013-04-16 2023-12-12 Regeneron Pharmaceuticals, Inc. Targeted modification of rat genome
EP3597755A1 (en) 2013-06-17 2020-01-22 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
CA2915837A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
EP3674411A1 (en) 2013-06-17 2020-07-01 The Broad Institute, Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20240172759A (ko) 2013-06-17 2024-12-10 더 브로드 인스티튜트, 인코퍼레이티드 간의 표적화 및 치료를 위한 ＣＲＩＳＰＲ­Ｃａｓ 시스템, 벡터 및 조성물의 전달 및 용도
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
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
EP3038661B1 (en) 2013-08-29 2023-12-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
SG10201700961TA (en) 2013-12-11 2017-04-27 Regeneron Pharma Methods and compositions for the targeted modification of a genome
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3653229A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CN105899658B (zh) * 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089354A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
KR20160130392A (ko) * 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
BR112016028023A2 (pt) 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
EP3155101B1 (en) 2014-06-16 2020-01-29 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas using the h1 promoter
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
DK3207124T3 (da) 2014-10-15 2019-08-12 Regeneron Pharma Fremgangsmåder og sammensætninger til generering eller bevaring af pluripotente celler
EP3221457B1 (en) 2014-11-21 2019-03-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modification using paired guide rnas
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology 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
CA3000187A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
CN108136047B (zh) * 2015-05-13 2022-12-13 西雅图儿童医院（Dba西雅图儿童研究所） 提高原代细胞中基于核酸内切酶的基因编辑
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
JP2018516596A (ja) * 2015-05-29 2018-06-28 アジェノビア コーポレーション 抗ウイルスの方法および組成物
US20160346360A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
MX2017015582A (es) * 2015-06-01 2018-09-06 Univ Temple Metodos y composiciones para tratamiento guiado por arn de infeccion por vih.
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
MX392008B (es) 2015-06-18 2025-03-21 Broad Inst Inc Mutaciones de la enzima crispr que reducen los efectos fuera del blanco
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
WO2017040348A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
CA2999912A1 (en) * 2015-09-28 2017-04-06 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
WO2017058888A1 (en) * 2015-10-02 2017-04-06 Agenovir Corporation Transgenic nuclease systems and methods
CN108513580A (zh) 2015-10-08 2018-09-07 哈佛学院董事及会员团体 多重基因组编辑
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
EP4434589A3 (en) 2015-10-23 2025-05-14 President and Fellows of Harvard College Evolved cas9 proteins for 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
JP2019506156A (ja) * 2016-01-25 2019-03-07 エクシジョン バイオセラピューティクス インコーポレイテッド Ｈｉｖ感染症のｒｎａ誘導型治療のための方法及び組成物
WO2017142835A1 (en) * 2016-02-15 2017-08-24 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
EP3420089B1 (en) * 2016-02-26 2021-12-29 LanzaTech NZ, Inc. 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
JP2019519250A (ja) * 2016-05-10 2019-07-11 ユナイテッド ステイツ ガバメント アズ リプレゼンテッド バイ ザ デパートメント オブ ベテランズ アフェアーズＵｎｉｔｅｄ Ｓｔａｔｅｓ Ｇｏｖｅｒｎｍｅｎｔ Ａｓ Ｒｅｐｒｅｓｅｎｔｅｄ Ｂｙ Ｔｈｅ Ｄｅｐａｒｔｍｅｎｔ Ｏｆ Ｖｅｔｅｒａｎｓ Ａｆｆａｉｒｓ Ｈｉｖ−１感染と複製に必須な遺伝子を切断するｃｒｉｓｐｒ／ｃａｓの構築物のレンチウィルスによる送達
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
CN109475109B (zh) 2016-05-20 2021-10-29 瑞泽恩制药公司 用于使用多个引导rna来破坏免疫耐受性的方法
CA3018294A1 (en) * 2016-06-01 2017-12-07 Excision Biotherapeutics, Inc. Compositions and methods of treatment for lytic and lysogenic viruses
CA3026332A1 (en) * 2016-06-03 2017-12-14 Temple University - Of The Commonwealth System Of Higher Education Negative feedback regulation of hiv-1 by gene editing strategy
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
US11661590B2 (en) 2016-08-09 2023-05-30 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
CA3034410A1 (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
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
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
US11898179B2 (en) 2017-03-09 2024-02-13 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
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
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
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
BR112019021719A2 (pt) 2017-04-21 2020-06-16 The General Hospital Corporation Variantes de cpf1 (cas12a) com especificidade para pam alterada
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
EP3630970A4 (en) 2017-05-25 2020-12-30 The General Hospital Corporation BASIC EDITORS WITH IMPROVED PRECISION AND SPECIFICITY
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
KR20200121782A (ko) 2017-10-16 2020-10-26 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US11618780B2 (en) 2017-10-20 2023-04-04 City Of Hope Composition and method for activating latent human immunodeficiency virus (HIV)
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020160418A1 (en) * 2019-01-31 2020-08-06 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
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220018504A (ko) 2019-05-23 2022-02-15 크리스티아나 케어 헬스 서비시즈 인코포레이티드 암 치료를 위한 변이체 nrf2의 유전자 녹아웃
US12203070B2 (en) 2019-05-23 2025-01-21 Christiana Care Gene Editing Institute, Inc. Gene knockout of NRF2 for treatment of cancer
WO2021072329A1 (en) * 2019-10-09 2021-04-15 Bluerock Therapeutics Lp Cells with sustained transgene expression
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021216503A1 (en) 2020-04-20 2021-10-28 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
EP4179120A4 (en) * 2020-07-13 2024-08-21 The United States Government as represented by the Department of Veterans Affairs Methods and compositions for crispr/cas9 guide rna efficiency and specificity against genetically diverse hiv-1 isolates
JP2023553422A (ja) * 2020-12-01 2023-12-21 ユナイテッド ステイツ ガバメント アズ リプレゼンテッド バイ ザ デパートメント オブ ベテランズ アフェアーズ ウイルスゲノムを切断するための組成物及び方法
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
US20250018060A1 (en) 2021-11-19 2025-01-16 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
CA3243905A1 (en) 2022-02-10 2023-08-17 Christiana Care Gene Editing Institute, Inc. METHODS FOR ADMINISTRATING A LIPID NANOPARTICLE FROM A 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
US20250001010A1 (en) 2023-06-30 2025-01-02 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
WO2025213016A2 (en) 2024-04-04 2025-10-09 Christiana 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
WO2026006542A2 (en) 2024-06-26 2026-01-02 Yale University Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome

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
EP0745134A1 (en) 1994-02-22 1996-12-04 Danafarber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
EP0915969A4 (en) * 1996-04-02 2002-04-17 Progenics Pharm Inc SUPPRESSION OF CD4 + CELL INFECTION BY HIV
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
WO2014093622A2 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
PT2898075E (pt) 2012-12-12 2016-06-16 Harvard College Manipulação e otimização de sistemas, métodos e composições de enzima melhorados para manipulação de sequências
EP4282970A3 (en) 2012-12-17 2024-01-17 President and Fellows of Harvard College Rna-guided human genome engineering
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
EP3038661B1 (en) 2013-08-29 2023-12-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
BR112016028023A2 (pt) * 2014-05-30 2017-08-22 Univ Leland Stanford Junior Composições e métodos de administração de tratamentos para infecções virais latentes
MX2017015582A (es) 2015-06-01 2018-09-06 Univ Temple Metodos y composiciones para tratamiento guiado por arn de infeccion por vih.
CA2999912A1 (en) 2015-09-28 2017-04-06 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
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
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
JP2019506156A (ja) 2016-01-25 2019-03-07 エクシジョン バイオセラピューティクス インコーポレイテッド Ｈｉｖ感染症のｒｎａ誘導型治療のための方法及び組成物
WO2017142835A1 (en) 2016-02-15 2017-08-24 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
US20190093091A1 (en) 2016-04-06 2019-03-28 Temple University - Of The Commonwealth System Of Higher Education Compositions for eradicating flavivirus infections in subjects
US20190093092A1 (en) 2016-05-05 2019-03-28 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
US11368243B2 (en) 2016-05-26 2022-06-21 Nokia Technologies Oy Codeword adaptation for non-orthogonal coded access
US20190256844A1 (en) 2016-06-07 2019-08-22 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
WO2018071623A2 (en) 2016-10-12 2018-04-19 Temple University - Of The Commonwealth System Of Higher Education Combination therapies for eradicating flavivirus infections in subjects
US20180208914A1 (en) 2017-01-26 2018-07-26 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

Also Published As

Publication number Publication date
US20180236043A1 (en) 2018-08-23
JP2023071897A (ja) 2023-05-23
US9981020B2 (en) 2018-05-29
JP2020188757A (ja) 2020-11-26
IL244220A0 (en) 2016-04-21
EP3038661A1 (en) 2016-07-06
SG11201601313TA (en) 2016-03-30
US20180169193A1 (en) 2018-06-21
WO2015031775A1 (en) 2015-03-05
AU2020264257A1 (en) 2020-11-26
US11285193B2 (en) 2022-03-29
AU2020264257B2 (en) 2022-10-20
ZA201601344B (en) 2017-05-31
CN106102781A (zh) 2016-11-09
US9925248B2 (en) 2018-03-27
KR20160060659A (ko) 2016-05-30
KR102829712B1 (ko) 2025-07-10
US20180236042A1 (en) 2018-08-23
US20180200343A1 (en) 2018-07-19
US20210252114A1 (en) 2021-08-19
US20180169194A1 (en) 2018-06-21
IL244220B (en) 2020-06-30
SG10201801658XA (en) 2018-03-28
US20180303915A1 (en) 2018-10-25
EP4385567A3 (en) 2024-08-14
US20180207243A1 (en) 2018-07-26
EP3038661A4 (en) 2017-04-05
US20160017301A1 (en) 2016-01-21
US20180228876A1 (en) 2018-08-16
US20180236046A1 (en) 2018-08-23
US20210069303A1 (en) 2021-03-11
JP7118588B2 (ja) 2022-08-16
MX2016002586A (es) 2016-06-14
JP2016534125A (ja) 2016-11-04
US20180228875A1 (en) 2018-08-16
US20180236044A1 (en) 2018-08-23
CA2922428A1 (en) 2015-03-05
US20240139294A1 (en) 2024-05-02
US20180228874A1 (en) 2018-08-16
US20180236045A1 (en) 2018-08-23
EP3038661B1 (en) 2023-12-27
EA037850B1 (ru) 2021-05-27
EA201600226A1 (ru) 2016-09-30
US11291710B2 (en) 2022-04-05
US20180221458A1 (en) 2018-08-09
US20220313795A1 (en) 2022-10-06
EP4385567A2 (en) 2024-06-19
US11298411B2 (en) 2022-04-12
US20230181698A1 (en) 2023-06-15
US20180169195A1 (en) 2018-06-21
US20160250300A1 (en) 2016-09-01
US20180214521A1 (en) 2018-08-02
US20180236041A1 (en) 2018-08-23
AU2014312123A1 (en) 2016-03-17
KR20230017364A (ko) 2023-02-03
BR112016004091A2 (pt) 2017-10-17

Similar Documents

Publication Publication Date Title
CL2016000424A1 (es) Métodos y composiciones para el arn-guiado tratamiento de la infección por vih
PT3785717T (pt) Métodos para o tratamento de infeções por coronaviridae
PT3019503T (pt) Compostos carbamoílpiridona- policíclicos e seu uso para o tratamento de infecções por hiv
SI3019499T1 (sl) Policiklične karbamoilpiridonske spojine in njihova uporaba za zdravljenje HIV infekcij
LT2984166T (lt) Kompozicijos, skirtos mpsi gydymui
EP3164406C0 (en) BORONIC ACID DERIVATIVES AND THEIR THERAPEUTIC USES
CL2016003071A1 (es) Composiciones y métodos para tratar y prevenir infecciones por staphylococcus aureus
LT3295942T (lt) Derinys, apimantis 5-fenoksi-3h-pirimidin-ono darinius ir jų panaudojimas živ infekcijos profikatikai ir gydymui
PL3277270T3 (pl) Kompozycje i sposoby leczenia niedokrwistości
LT3426226T (lt) Oftalmologinė kompozicija
PT2859896T (pt) Composições farmacêuticas para o tratamento de distúrbios musculares
EP3012292A4 (en) PROCESSING AGENTS AND COMPOSITION
ZA201702884B (en) Compositions and methods for the treatment and prophylaxis of surgical site infections
CL2014001883A1 (es) Compuestos y métodos para el tratamiento de las infecciones de candidiasis y aspergillus
LT2939665T (lt) Farmacinė kompozicija, skirta živ infekcijų gydymui
PL3016663T3 (pl) Sterylizowana kompozycja termożelowalna
PL3145592T3 (pl) Kompozycja do pielęgnacji skóry
PL3373922T3 (pl) Kompozycje i sposoby do stosowania do leczenia homocystynurii
DK3233104T3 (da) Immunterapi-behandlinger og -sammensætninger
IL256919A (en) Pharmaceutical compositions useful for the treatment of tissue injury
MA43166A (fr) Formulation de fviii
LT3096839T (lt) Kvapnios kompozicijos kūnui
LT3015109T (lt) Biologinis barjeras su simetikonu, skirtas panaudoti nosies-ryklės-vamzdelių infekcijų gydymui
TH1501005357A (th) สารประกอบพิแรโซล-เอไมด์ และการใช้ทางการแพทย์ของมัน
ES1113580Y (es) Dispositivo dental para el tratamiento de la onicofagia