BR112016004091A2 - métodos e composições para tratamento guiado por rna de infecção por hiv - Google Patents

métodos e composições para tratamento guiado por rna de infecção por hiv

Info

Publication number
BR112016004091A2
BR112016004091A2 BR112016004091A BR112016004091A BR112016004091A2 BR 112016004091 A2 BR112016004091 A2 BR 112016004091A2 BR 112016004091 A BR112016004091 A BR 112016004091A BR 112016004091 A BR112016004091 A BR 112016004091A BR 112016004091 A2 BR112016004091 A2 BR 112016004091A2
Authority
BR
Brazil
Prior art keywords
compositions
methods
hiv infection
rna
guided treatment
Prior art date
Application number
BR112016004091A
Other languages
English (en)
Inventor
Khalili Kamel
Hu Wenhui
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 BR112016004091A2 publication Critical patent/BR112016004091A2/pt

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 RNAses, DNAses
    • 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 [CRISPRs]
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Chemical Kinetics & Catalysis (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Developmental Biology & Embryology (AREA)
  • Urology & Nephrology (AREA)
  • Cell Biology (AREA)
  • AIDS & HIV (AREA)

Abstract

resumo métodos e composições para tratamento guiado por rna de infecção por hiv a presente invenção apresenta métodos e composições para o tratamento da infecção pelo vírus da imunodeficiência. as composições incluem sequências de ácidos nucleicos isoladas compreendendo uma endonuclease associada-crispr de arn e um guia, em que o arn guia é complementar a uma sequência alvo de um vírus da imunodeficiência humana.
BR112016004091A 2013-08-29 2014-08-29 métodos e composições para tratamento guiado por rna de infecção por hiv BR112016004091A2 (pt)

Applications Claiming Priority (4)

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
PCT/US2014/053441 WO2015031775A1 (en) 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection

Publications (1)

Publication Number Publication Date
BR112016004091A2 true BR112016004091A2 (pt) 2017-10-17

Family

ID=52587370

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016004091A BR112016004091A2 (pt) 2013-08-29 2014-08-29 métodos e composições para tratamento guiado por rna de infecção por hiv

Country Status (15)

Country Link
US (24) US9925248B2 (pt)
EP (2) EP3038661B1 (pt)
JP (3) JP7118588B2 (pt)
KR (2) KR20160060659A (pt)
CN (1) CN106102781A (pt)
AU (2) AU2014312123A1 (pt)
BR (1) BR112016004091A2 (pt)
CA (1) CA2922428A1 (pt)
CL (1) CL2016000424A1 (pt)
EA (1) EA037850B1 (pt)
IL (1) IL244220B (pt)
MX (1) MX2016002586A (pt)
SG (2) SG10201801658XA (pt)
WO (1) WO2015031775A1 (pt)
ZA (1) ZA201601344B (pt)

Families Citing this family (99)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2883590T3 (es) 2012-12-12 2021-12-09 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas
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
WO2014113493A1 (en) 2013-01-16 2014-07-24 Emory University Cas9-nucleic acid complexes and uses related thereto
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
EP3741868B1 (en) 2013-03-15 2024-05-22 The General Hospital Corporation Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
KR20200136508A (ko) 2013-04-16 2020-12-07 리제너론 파마슈티칼스 인코포레이티드 랫트 게놈의 표적화된 변형
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
JP6665088B2 (ja) 2013-06-17 2020-03-13 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
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
BR112016004091A2 (pt) 2013-08-29 2017-10-17 Univ Temple métodos e composições para tratamento guiado por rna de infecção por hiv
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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
MX2016007654A (es) 2013-12-11 2017-08-15 Regeneron Pharma Metodos y composiciones para la modificacion dirigida de un genoma.
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
CA2932436A1 (en) 2013-12-12 2015-06-18 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
MX2016007324A (es) * 2013-12-12 2017-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.
KR20160089527A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
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
US10286084B2 (en) * 2014-02-18 2019-05-14 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
KR20170005494A (ko) 2014-05-30 2017-01-13 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법
EP3674408A1 (en) 2014-06-16 2020-07-01 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3207124B1 (en) 2014-10-15 2019-06-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
NZ731962A (en) 2014-11-21 2022-07-01 Regeneron Pharma Methods and compositions for targeted genetic modification using paired guide rnas
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
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
EP3300507A4 (en) * 2015-04-02 2019-03-13 Agenovir Corporation GENERIC ADMINISTRATION AND COMPOSITIONS
JP2018520648A (ja) * 2015-05-13 2018-08-02 シアトル チルドレンズ ホスピタル， ディービーエー シアトル チルドレンズ リサーチ インスティテュート 初代細胞におけるエンドヌクレアーゼに基づいた遺伝子編集の向上
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
CA3000170A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Antiviral methods and compositions
WO2016196282A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Compositions and methods for cell targeted hpv treatment
AU2016270702B2 (en) * 2015-06-01 2022-01-20 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
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
CA2996888A1 (en) 2015-08-28 2017-03-09 The General Hospital Corporation Engineered crispr-cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 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
JP7059468B2 (ja) * 2015-10-08 2022-04-26 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 多重ゲノム編集
EP3362104A4 (en) * 2015-10-16 2019-03-27 Temple University - Of The Commonwealth System of Higher Education METHODS AND COMPOSITIONS USING CPF1 FOR RNA-GUIDED GENETIC EDITION
EP4269577A3 (en) 2015-10-23 2024-01-17 President and Fellows of Harvard College Nucleobase editors and uses thereof
RU2018124657A (ru) 2015-12-09 2020-01-09 Эксижн Биотерапевтикс, Инк. Способы редактирования генов и композиции для устранения риска активации вируса jc и пмл (прогрессирующая мультифокальная лейкоэнцефалопатия) во время иммуносупрессивной терапии
EP3407918A4 (en) * 2016-01-25 2019-09-04 Excision Biotherapeutics METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
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
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
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
IL264565B1 (en) 2016-08-03 2024-03-01 Harvard College Adenosine nuclear base editors and their uses
EP3497214B1 (en) 2016-08-09 2023-06-28 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
CN109689866A (zh) * 2016-09-12 2019-04-26 切除生物治疗公司 Hiv临床计划
KR101997116B1 (ko) * 2016-10-14 2019-07-05 연세대학교 산학협력단 Kras 유전자에 상보적인 가이드 rna 및 이의 용도
SG11201903089RA (en) 2016-10-14 2019-05-30 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
US10745677B2 (en) 2016-12-23 2020-08-18 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
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
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
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018194876A1 (en) * 2017-04-17 2018-10-25 Temple University- Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
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
CN111093714A (zh) 2017-05-25 2020-05-01 通用医疗公司 使用分割型脱氨酶限制不需要的脱靶碱基编辑器脱氨
EP3658573A1 (en) 2017-07-28 2020-06-03 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
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
US11618780B2 (en) 2017-10-20 2023-04-04 City Of Hope Composition and method for activating latent human immunodeficiency virus (HIV)
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation ADENINE DNA BASE EDITOR VARIANTS WITH REDUCED OFF-TARGET RNA EDITING
WO2020191246A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020237186A1 (en) 2019-05-23 2020-11-26 Christiana Care Health Services, Inc. Gene knockout of nrf2 for treatment of cancer
KR20220018504A (ko) 2019-05-23 2022-02-15 크리스티아나 케어 헬스 서비시즈 인코포레이티드 암 치료를 위한 변이체 nrf2의 유전자 녹아웃
US20230257771A1 (en) 2020-04-20 2023-08-17 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
GB2614813A (en) 2020-05-08 2023-07-19 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
WO2022015702A2 (en) * 2020-07-13 2022-01-20 Howell Alexandra L Methods and compositions for crispr/cas9 guide rna efficiency and specificity against genetically diverse hiv-1 isolates
CA3200929A1 (en) * 2020-12-01 2022-06-09 Alexandra HOWELL Compositions and methods for cleaving viral genomes
CA3207527A1 (en) 2021-02-05 2022-08-11 Eric B. Kmiec Methods of and compositions for reducing gene expression and/or activity
CA3238629A1 (en) 2021-11-19 2023-05-25 Christiana Care Gene Editing Institute, Inc. Methods for intratumoral delivery of crispr/cas systems
WO2023091696A1 (en) 2021-11-19 2023-05-25 Christiana Care Gene Editing Institute, Inc. Adenovirus delivery system for cancer treatment
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
WO2023154451A1 (en) 2022-02-10 2023-08-17 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

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
CA2183667A1 (en) 1994-02-22 1995-08-24 Wayne A. Marasco Nucleic acid delivery system, method of synthesis and uses thereof
AU728512B2 (en) * 1996-04-02 2001-01-11 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
EP1849873B1 (en) * 1999-04-29 2011-10-12 Gbp Ip, Llc 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
ES2883590T3 (es) 2012-12-12 2021-12-09 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas
DK2898075T3 (en) 2012-12-12 2016-06-27 Broad Inst Inc CONSTRUCTION AND OPTIMIZATION OF IMPROVED SYSTEMS, PROCEDURES AND ENZYME COMPOSITIONS FOR SEQUENCE MANIPULATION
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
WO2014099750A2 (en) 2012-12-17 2014-06-26 President And Fellows Of Harvard College Rna-guided human genome engineering
CA2908253C (en) * 2013-04-04 2024-01-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
BR112016004091A2 (pt) 2013-08-29 2017-10-17 Univ Temple métodos e composições para tratamento guiado por rna de infecção por hiv
KR20170005494A (ko) * 2014-05-30 2017-01-13 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법
AU2016270702B2 (en) 2015-06-01 2022-01-20 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for RNA-guided treatment of HIV infection
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
EP3362104A4 (en) 2015-10-16 2019-03-27 Temple University - Of The Commonwealth System of Higher Education METHODS AND COMPOSITIONS USING CPF1 FOR RNA-GUIDED GENETIC EDITION
RU2018124657A (ru) 2015-12-09 2020-01-09 Эксижн Биотерапевтикс, Инк. Способы редактирования генов и композиции для устранения риска активации вируса jc и пмл (прогрессирующая мультифокальная лейкоэнцефалопатия) во время иммуносупрессивной терапии
EP3407918A4 (en) 2016-01-25 2019-09-04 Excision Biotherapeutics METHOD AND COMPOSITIONS FOR RNA-TREATED TREATMENT OF HIV INFECTIONS
WO2018106268A1 (en) 2016-01-25 2018-06-14 Excision Biotherapeutics Rna guided eradication of human jc virus and other polyomaviruses
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
US20190093092A1 (en) 2016-05-05 2019-03-28 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 ADAPTATION OF CODE WORD 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
JP2020505390A (ja) 2017-01-26 2020-02-20 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 Ｃｒｉｓｐｒ治療薬を送達するためのウイルスベクターとしてのレンチウイルスおよび非組み込みレンチウイルス
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
WO2018194876A1 (en) 2017-04-17 2018-10-25 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
US20180228876A1 (en) 2018-08-16
KR20160060659A (ko) 2016-05-30
US20180228874A1 (en) 2018-08-16
US20180200343A1 (en) 2018-07-19
IL244220B (en) 2020-06-30
IL244220A0 (en) 2016-04-21
JP2023071897A (ja) 2023-05-23
US20180236045A1 (en) 2018-08-23
WO2015031775A1 (en) 2015-03-05
EP3038661A1 (en) 2016-07-06
AU2014312123A1 (en) 2016-03-17
CA2922428A1 (en) 2015-03-05
EP4385567A2 (en) 2024-06-19
US20180236046A1 (en) 2018-08-23
AU2020264257A1 (en) 2020-11-26
US20180169195A1 (en) 2018-06-21
EA037850B1 (ru) 2021-05-27
US20180214521A1 (en) 2018-08-02
US11291710B2 (en) 2022-04-05
JP7118588B2 (ja) 2022-08-16
SG10201801658XA (en) 2018-03-28
US20180207243A1 (en) 2018-07-26
SG11201601313TA (en) 2016-03-30
EP3038661B1 (en) 2023-12-27
US20180169194A1 (en) 2018-06-21
CL2016000424A1 (es) 2017-07-14
JP2020188757A (ja) 2020-11-26
US11298411B2 (en) 2022-04-12
KR20230017364A (ko) 2023-02-03
CN106102781A (zh) 2016-11-09
US20180236042A1 (en) 2018-08-23
JP2016534125A (ja) 2016-11-04
AU2020264257B2 (en) 2022-10-20
US20210252114A1 (en) 2021-08-19
EA201600226A1 (ru) 2016-09-30
US20180169193A1 (en) 2018-06-21
US20180236044A1 (en) 2018-08-23
ZA201601344B (en) 2017-05-31
US11285193B2 (en) 2022-03-29
EP3038661A4 (en) 2017-04-05
US20180303915A1 (en) 2018-10-25
US9925248B2 (en) 2018-03-27
MX2016002586A (es) 2016-06-14
US20210069303A1 (en) 2021-03-11
US20180228875A1 (en) 2018-08-16
US20240139294A1 (en) 2024-05-02
US20180221458A1 (en) 2018-08-09
US9981020B2 (en) 2018-05-29
US20180236043A1 (en) 2018-08-23
US20180236041A1 (en) 2018-08-23
US20160017301A1 (en) 2016-01-21
US20230181698A1 (en) 2023-06-15
US20160250300A1 (en) 2016-09-01
US20220313795A1 (en) 2022-10-06

Similar Documents

Publication Publication Date Title
BR112016004091A2 (pt) métodos e composições para tratamento guiado por rna de infecção por hiv
PH12020551055A1 (en) Methods for treating filoviridae virus infections
PH12018500571A1 (en) Hepatitis b core protein modulators
CL2018001056A1 (es) Vacuna contra el virus herpes simplex
MX2019003525A (es) Moduladores alostericos de proteina nucleo de hepatitis b.
BR112016023558A2 (pt) compostos úteis como imunomoduladores
BR112018013065A2 (pt) composições e métodos para o tratamento de hemoglobinopatias
EA201890012A1 (ru) Способы и композиции для рнк-направляемого лечения инфекции, вызываемой вич
SA516370684B1 (ar) مثبطات بوليمراز فيروس الالتهاب الكبدي سي
NZ630800A (en) Methods of preparing substituted nucleotide analogs
EA201491953A1 (ru) СОЕДИНЕНИЯ НА ОСНОВЕ МИКРОРНК И СПОСОБЫ МОДУЛИРОВАНИЯ АКТИВНОСТИ miR-21
UY35873A (es) ?composiciones y métodos para el control de virus en el ácaro varroa y las abejas?.
UY36868A (es) Composiciones para tratamiento de enfermedades mediadas por interleuquina 5 (il-5)
GT201600066A (es) Composiciones y métodos para inhibir la expresión del gen alas1
BR112017003663A2 (pt) composto, composição, e, método para tratamento de infecção por hiv.
MA40220A (fr) Pyridinones substitués par phényle et acide tertbutylacétique à effet anti-vih
PE20160596A1 (es) Formulacion de atazanavir y cobicistat para el tratamiento del virus de inmunodeficiencia humana
CL2018000015A1 (es) Moléculas de fusión
BR112014028632A8 (pt) Composto, composição farmacêutica, e, método para preparar um composto
EP3765048A4 (en) USE OF INFECTIOUS NUCLEIC ACID TO TREAT CANCER
BR112016020895A2 (pt) moduladores alostéricos da proteína do núcleo da hepatite b
EA201990021A1 (ru) Способы лечения вирусных инфекций filoviridae
EA201691835A1 (ru) Аллостерические модуляторы капсидного белка гепатита в

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements
B350 Update of information on the portal [chapter 15.35 patent gazette]