[go: up one dir, main page]

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

    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0034Urogenital system, e.g. vagina, uterus, cervix, penis, scrotum, urethra, bladder; Personal lubricants
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)

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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Reproductive Health (AREA)
  • Urology & Nephrology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Tropical Medicine & Parasitology (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) KR20160060659A (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) SG10201801658XA (es)
WO (1) WO2015031775A1 (es)
ZA (1) ZA201601344B (es)

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 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
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
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CN113005148A (zh) 2013-01-16 2021-06-22 爱默蕾大学 Cas9-核酸复合物及其相关用途
KR102271292B1 (ko) 2013-03-15 2021-07-02 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
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
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
MX369747B (es) 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
BR122021009076B1 (pt) 2013-06-17 2024-02-15 The Broad Institute Inc. Vetor viral contendo molécula(s) de ácido nucleico heterólogo, composição, uso e métodos do mesmo
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
AU2014281026B2 (en) 2013-06-17 2020-05-28 Massachusetts Institute Of Technology Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
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
US9925248B2 (en) * 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
CN111218447B (zh) 2013-11-07 2024-10-11 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
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
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
EP3653703A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
WO2015089462A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
SG10201804975PA (en) * 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
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
KR20160130392A (ko) * 2014-02-18 2016-11-11 듀크 유니버시티 바이러스 복제의 불활성화를 위한 조성물 및 그의 제조 및 사용 방법
AU2015266776A1 (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
AU2015277369B2 (en) 2014-06-16 2021-08-19 The Johns Hopkins University Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
PT3207124T (pt) 2014-10-15 2019-08-27 Regeneron Pharma Métodos e composições para a produção ou conservação de células pluripotentes
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
EP3985115A1 (en) 2014-12-12 2022-04-20 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
WO2016161260A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
CA2985650A1 (en) 2015-05-13 2016-11-17 Seattle Children's Hospital (dba Seattle Children's Research Institute) Enhancing endonuclease based gene editing in primary cells
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
US20160350476A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Antiviral methods and compositions
WO2016196282A1 (en) * 2015-05-29 2016-12-08 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
JP7107683B2 (ja) 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるcrispr酵素突然変異
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
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
HK1257676A1 (zh) 2015-08-28 2019-10-25 The General Hospital Corporation 工程化 crispr-cas 9 核酸酶
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
EP3356521A4 (en) * 2015-09-28 2019-03-13 Temple University - Of The Commonwealth System of Higher Education METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
US20170096649A1 (en) * 2015-10-02 2017-04-06 Agenovir Corporation Transgenic nuclease systems and methods
MX2018004263A (es) * 2015-10-08 2019-08-16 Harvard College Edición genómica multiplexada.
JP2018531261A (ja) * 2015-10-16 2018-10-25 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 Cpf1を用いた、rnaガイド遺伝子編集方法および組成物
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
JP2019508364A (ja) 2015-12-09 2019-03-28 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 免疫抑制療法中のjcウイルス活性化およびpml(進行性多巣性白質脳症)のリスクを除去するための遺伝子編集方法および組成物
US20190032057A1 (en) * 2016-01-25 2019-01-31 Excision Biotherapeutics, Inc. Methods and compositions for rna-guided treatment of hiv infection
WO2017142835A1 (en) * 2016-02-15 2017-08-24 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
KR20180110144A (ko) * 2016-02-26 2018-10-08 란자테크 뉴질랜드 리미티드 C1-고정 박테리아에 대한 crispr/cas 시스템
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US20190225956A1 (en) * 2016-05-10 2019-07-25 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
EP4368637A3 (en) 2016-05-20 2024-07-10 Regeneron Pharmaceuticals, Inc. Methods for breaking immunological tolerance using multiple guide rnas
WO2017210380A1 (en) * 2016-06-01 2017-12-07 Excision Biotherapeutics, Inc. Compositions and methods of treatment for lytic and lysogenic viruses
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
GB2568182A (en) 2016-08-03 2019-05-08 Harvard College Adenosine nucleobase editors and uses thereof
WO2018031683A1 (en) 2016-08-09 2018-02-15 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
AU2017322697A1 (en) * 2016-09-12 2019-03-07 Excision Biotherapeutics Inc HIV clinical plan
WO2018070850A1 (ko) * 2016-10-14 2018-04-19 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
KR101997116B1 (ko) 2016-10-14 2019-07-05 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
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
US20190367924A1 (en) * 2017-02-17 2019-12-05 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
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
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
US11268082B2 (en) 2017-03-23 2022-03-08 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
KR102746223B1 (ko) 2017-04-20 2024-12-27 이제네시스, 인크. 유전자 변형 동물의 생성 방법
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
JP2020521451A (ja) 2017-05-25 2020-07-27 ザ ジェネラル ホスピタル コーポレイション 望ましくないオフターゲット塩基エディター脱アミノ化を制限するためのスプリットデアミナーゼの使用
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
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
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
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
JP7585237B2 (ja) 2019-05-23 2024-11-18 クリスティアナ ケア ジーン エディティング インスティテュート,インコーポレーテッド がんを処置するためのバリアントnrf2の遺伝子ノックアウト
CN114127291A (zh) 2019-05-23 2022-03-01 克里斯蒂安娜保健服务公司 用于治疗癌症的nrf2基因敲除
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
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
MX2023000661A (es) * 2020-07-13 2023-07-03 Alexandra L Howell Métodos y composiciones para la eficiencia y especificidad de arn guía de crispr/cas9 frente a aislados de vih-1 genéticamente diferentes.
CA3200929A1 (en) * 2020-12-01 2022-06-09 Alexandra HOWELL 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
US20250018060A1 (en) 2021-11-19 2025-01-16 Christiana Care Gene Editing Institute, Inc. Adenovirus delivery system for cancer treatment
AU2022393213A1 (en) 2021-11-19 2024-05-30 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
EP4476342A1 (en) 2022-02-10 2024-12-18 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
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

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
AU778698B2 (en) * 1999-04-29 2004-12-16 Miltenyi Biotec B.V. & Co. KG 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
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
EP2898075B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
JP6700788B2 (ja) 2012-12-17 2020-05-27 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 Rna誘導性ヒトゲノム改変
CA2908253C (en) 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
US9925248B2 (en) 2013-08-29 2018-03-27 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
AU2015266776A1 (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
MX2017015582A (es) 2015-06-01 2018-09-06 Univ Temple Metodos y composiciones para tratamiento guiado por arn de infeccion por vih.
EP3356521A4 (en) 2015-09-28 2019-03-13 Temple University - Of The Commonwealth System of Higher Education METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
JP2018531261A (ja) 2015-10-16 2018-10-25 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 Cpf1を用いた、rnaガイド遺伝子編集方法および組成物
JP2019508364A (ja) 2015-12-09 2019-03-28 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 免疫抑制療法中のjcウイルス活性化およびpml(進行性多巣性白質脳症)のリスクを除去するための遺伝子編集方法および組成物
JP2019512458A (ja) 2016-01-25 2019-05-16 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 Rnaによって誘導された、ヒトjcウイルス及び他のポリオーマウイルスの根絶
US20190032057A1 (en) 2016-01-25 2019-01-31 Excision Biotherapeutics, Inc. Methods and compositions for rna-guided treatment of hiv infection
WO2017142835A1 (en) 2016-02-15 2017-08-24 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
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
CN110431230A (zh) 2017-01-26 2019-11-08 切除生物治疗公司 慢病毒和非整合型慢病毒作为病毒载体实施crispr治疗
US20190367924A1 (en) 2017-02-17 2019-12-05 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
US11298411B2 (en) 2022-04-12
BR112016004091A2 (pt) 2017-10-17
EA037850B1 (ru) 2021-05-27
US20180303915A1 (en) 2018-10-25
US20180169193A1 (en) 2018-06-21
US20180236045A1 (en) 2018-08-23
IL244220B (en) 2020-06-30
EP3038661B1 (en) 2023-12-27
EP3038661A1 (en) 2016-07-06
JP2016534125A (ja) 2016-11-04
US20180169195A1 (en) 2018-06-21
EP4385567A2 (en) 2024-06-19
US20240139294A1 (en) 2024-05-02
US20160250300A1 (en) 2016-09-01
SG11201601313TA (en) 2016-03-30
KR20230017364A (ko) 2023-02-03
US20180236044A1 (en) 2018-08-23
US20210252114A1 (en) 2021-08-19
JP2020188757A (ja) 2020-11-26
EP3038661A4 (en) 2017-04-05
US20180236043A1 (en) 2018-08-23
US20180169194A1 (en) 2018-06-21
US9981020B2 (en) 2018-05-29
US11285193B2 (en) 2022-03-29
US20230181698A1 (en) 2023-06-15
US20180207243A1 (en) 2018-07-26
KR102829712B1 (ko) 2025-07-10
US20220313795A1 (en) 2022-10-06
US20180236046A1 (en) 2018-08-23
US20180221458A1 (en) 2018-08-09
US9925248B2 (en) 2018-03-27
AU2020264257A1 (en) 2020-11-26
JP2023071897A (ja) 2023-05-23
US20180228875A1 (en) 2018-08-16
EA201600226A1 (ru) 2016-09-30
US20180228876A1 (en) 2018-08-16
US20180236041A1 (en) 2018-08-23
US20180236042A1 (en) 2018-08-23
SG10201801658XA (en) 2018-03-28
EP4385567A3 (en) 2024-08-14
JP7118588B2 (ja) 2022-08-16
ZA201601344B (en) 2017-05-31
US20180214521A1 (en) 2018-08-02
MX2016002586A (es) 2016-06-14
AU2014312123A1 (en) 2016-03-17
CA2922428A1 (en) 2015-03-05
US20160017301A1 (en) 2016-01-21
US20210069303A1 (en) 2021-03-11
CN106102781A (zh) 2016-11-09
US20180228874A1 (en) 2018-08-16
US11291710B2 (en) 2022-04-05
AU2020264257B2 (en) 2022-10-20
US20180200343A1 (en) 2018-07-19
WO2015031775A1 (en) 2015-03-05
KR20160060659A (ko) 2016-05-30
IL244220A0 (en) 2016-04-21

Similar Documents

Publication Publication Date Title
CL2016000424A1 (es) Métodos y composiciones para el arn-guiado tratamiento de la infección por vih
SI3252058T1 (sl) Policiklične karbamoilpiridonske spojine in njihova uporaba za zdravljenje okužb s 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
HUE058903T2 (hu) Kombinációk, amelyek tartalmaznak 5-fenoxi-3H-pirimidin-4-on származékokat és felhasználásuk HIV infekció profilaxisára vagy kezelésére
CL2016003071A1 (es) Composiciones y métodos para tratar y prevenir infecciones por staphylococcus aureus
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
PL3145592T3 (pl) Kompozycja do pielęgnacji skóry
EP3471711A4 (en) ANTI-MALARIAL COMPOSITIONS AND THEIR USES
PL3373922T3 (pl) Kompozycje i sposoby do stosowania do leczenia homocystynurii
DK3233104T3 (da) Immunterapi-behandlinger og -sammensætninger
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
UA28257S (uk) Рукавички хірургічні
TH1601001266A (th) วิธีการและองค์ประกอบทางเภสัชกรรมสำหรับการบำบัดการติดเชื้อไวรัสตับอักเสบ b