[go: up one dir, main page]

SG10201801658XA - Methods and compositions for rna-guided treatment of hiv infection - Google Patents

Methods and compositions for rna-guided treatment of hiv infection

Info

Publication number
SG10201801658XA
SG10201801658XA SG10201801658XA SG10201801658XA SG10201801658XA SG 10201801658X A SG10201801658X A SG 10201801658XA SG 10201801658X A SG10201801658X A SG 10201801658XA SG 10201801658X A SG10201801658X A SG 10201801658XA SG 10201801658X A SG10201801658X A SG 10201801658XA
Authority
SG
Singapore
Prior art keywords
rna
compositions
methods
hiv infection
guided treatment
Prior art date
Application number
SG10201801658XA
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 SG10201801658XA publication Critical patent/SG10201801658XA/en

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)
  • 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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Reproductive Health (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Urology & Nephrology (AREA)
  • AIDS & HIV (AREA)
SG10201801658XA 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection SG10201801658XA (en)

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
SG10201801658XA true SG10201801658XA (en) 2018-03-28

Family

ID=52587370

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201801658XA SG10201801658XA (en) 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection
SG11201601313TA SG11201601313TA (en) 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201601313TA SG11201601313TA (en) 2013-08-29 2014-08-29 Methods and compositions for rna-guided treatment of hiv infection

Country Status (15)

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

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
ES2701749T3 (en) 2012-12-12 2019-02-25 Broad Inst Inc Methods, models, systems and apparatus to identify target sequences for Cas enzymes or CRISPR-Cas systems for target sequences and transmit results thereof
BR112015013784A2 (en) 2012-12-12 2017-07-11 Massachusetts Inst Technology application, manipulation 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
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
US9885033B2 (en) 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
SG10201808935WA (en) 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
DK3011031T3 (en) 2013-06-17 2020-12-21 Broad Inst Inc PERFORMANCE AND APPLICATION OF CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR LIVER TARGET DIRECTION AND THERAPY
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20250012194A (en) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
JP6625971B2 (en) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド Delivery, engineering and optimization of tandem guide systems, methods and compositions for array manipulation
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
KR102829712B1 (en) 2013-08-29 2025-07-10 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Methods and compositions for rna-guided treatment of hiv infection
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
CA2930015A1 (en) 2013-11-07 2015-05-14 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
JP6174811B2 (en) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Methods and compositions for targeted genomic modification
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
KR20160089530A (en) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
SG11201606819QA (en) * 2014-02-18 2016-09-29 Univ Duke Compositions for the inactivation of virus replication and methods of making and using the same
AU2015266770A1 (en) 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
SG11201610405RA (en) 2014-06-16 2017-01-27 Univ Johns Hopkins Compositions and methods for the expression of crispr guide rnas using the h1 promoter
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3561052A1 (en) 2014-10-15 2019-10-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
ES2731437T3 (en) 2014-11-21 2019-11-15 Regeneron Pharma Methods and compositions for directed genetic modification through the use of guide RNA pairs
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
MA41382A (en) * 2015-03-20 2017-11-28 Univ Temple GENE EDITING BASED ON THE TAT-INDUCED CRISPR / ENDONUCLEASE SYSTEM
CN104726449A (en) * 2015-03-23 2015-06-24 国家纳米科学中心 CRISPR-Cas9 system for preventing and/or treating HIV, as well as preparation method and application thereof
US20160287678A1 (en) * 2015-04-02 2016-10-06 Agenovir Corporation Gene delivery methods and compositions
US20180119174A1 (en) 2015-05-13 2018-05-03 Seattle Children's Hospita (dba Seattle Children's Research Institute Enhancing endonuclease based gene editing in primary cells
GB2543873A (en) * 2015-05-29 2017-05-03 Agenovir Corp Compositions and methods for cell targeted HPV treatment
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
WO2016196283A1 (en) * 2015-05-29 2016-12-08 Agenovir Corporation Antiviral methods and compositions
CA2987927C (en) * 2015-06-01 2024-03-19 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
CN104894071A (en) * 2015-06-08 2015-09-09 东华大学 Method for carrying out gene edition on GT1-7 cells
TWI813532B (en) 2015-06-18 2023-09-01 美商博得學院股份有限公司 Crispr enzyme mutations reducing off-target effects
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
KR102668608B1 (en) 2015-08-28 2024-05-24 더 제너럴 하스피탈 코포레이션 Engineered CRISPR-CAS9 nuclease
JP2018527943A (en) * 2015-09-28 2018-09-27 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション Methods and compositions for the treatment of RNA-induced HIV infection
WO2017058888A1 (en) * 2015-10-02 2017-04-06 Agenovir Corporation Transgenic nuclease systems and methods
NZ741778A (en) * 2015-10-08 2022-02-25 Harvard College Multiplexed genome editing
AU2016340078A1 (en) * 2015-10-16 2018-04-12 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions utilizing Cpf1 for RNA-guided gene editing
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and uses thereof
CA3006305A1 (en) 2015-12-09 2017-06-15 Excision Biotherapeutics, Inc. Gene editing methods and compositions for eliminating risk of jc virus activation and pml (progressive multifocal leukoencephalopathy) during immunosuppressive therapy
EP3407918A4 (en) * 2016-01-25 2019-09-04 Excision Biotherapeutics METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
AU2017219605B2 (en) * 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
SG11201807025SA (en) * 2016-02-26 2018-09-27 Lanzatech New Zealand Ltd Crispr/cas systems for c-1 fixing bacteria
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
CA3024494A1 (en) * 2016-05-10 2017-11-16 United States Government As Represented By The Department Of Veterans Affairs Lentiviral delivery of crispr/cas constructs that cleave genes essential for hiv-1 infection and replication
CN107365786A (en) * 2016-05-12 2017-11-21 中国科学院微生物研究所 A kind of method and its application being cloned into spacer sequences in CRISPR-Cas9 systems
MX2018014172A (en) 2016-05-20 2019-08-22 Regeneron Pharma Methods for breaking immunological tolerance using multiple guide rnas.
CA3018294A1 (en) * 2016-06-01 2017-12-07 Excision Biotherapeutics, Inc. Compositions and methods of treatment for lytic and lysogenic viruses
CN109640946A (en) * 2016-06-03 2019-04-16 天普大学-联邦高等教育系统 Negative feedback regulation of HIV-1 through gene editing strategies
KR20250103795A (en) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Adenosine nucleobase editors and uses thereof
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018049372A1 (en) * 2016-09-12 2018-03-15 Excision Biotherapeutics Inc Hiv clinical plan
KR101997116B1 (en) 2016-10-14 2019-07-05 연세대학교 산학협력단 Guide RNA complementary to KRAS gene and use thereof
JP7588390B2 (en) 2016-10-14 2024-11-22 プレジデント アンド フェローズ オブ ハーバード カレッジ AAV Delivery of Nucleobase Editors
WO2018070850A1 (en) * 2016-10-14 2018-04-19 연세대학교 산학협력단 Guide rna complementary to kras gene, and use thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US20190071673A1 (en) * 2017-01-18 2019-03-07 Thomas Malcolm CRISPRs WITH IMPROVED SPECIFICITY
WO2018152418A1 (en) * 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (en) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 Cancer vaccine
JP2020510439A (en) 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 Base-editing factor from cytosine to guanine
CN106755268B (en) * 2017-03-22 2020-07-17 中国科学院海洋研究所 Method for in vitro screening oyster active peptide with function of improving memory
BR112019019655A2 (en) 2017-03-23 2020-04-22 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
CN111031790A (en) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 Methods of producing genetically modified animals
CN110799525A (en) 2017-04-21 2020-02-14 通用医疗公司 Variants of CPF1 (CAS12a) with altered PAM specificity
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP7324713B2 (en) 2017-05-25 2023-08-10 ザ ジェネラル ホスピタル コーポレイション Base editor with improved accuracy and specificity
JP2020534795A (en) 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 Methods and Compositions for Evolving Base Editing Factors Using Phage-Supported Continuous Evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
KR20250107288A (en) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 Uses of adenosine base editors
WO2019079819A1 (en) * 2017-10-20 2019-04-25 City Of Hope Composition and method for activating latent human immunodeficiency virus (hiv)
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20220194990A1 (en) * 2019-01-31 2022-06-23 Board Of Regents Of The University Of Nebraska Virus-like particles and methods of use thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
EP3973056A1 (en) 2019-05-23 2022-03-30 Christiana Care Health Services, Inc. Gene knockout of variant nrf2 for treatment of cancer
WO2020237186A1 (en) 2019-05-23 2020-11-26 Christiana Care Health Services, Inc. Gene knockout of nrf2 for treatment of cancer
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US20230257771A1 (en) 2020-04-20 2023-08-17 Christiana Care Health Services, Inc. Aav delivery system for lung cancer treatment
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023534968A (en) * 2020-07-13 2023-08-15 アレクサンドラ エル ハウエル Methods and compositions for efficiency and specificity of CRISPR/CAS9 guide RNA against genetically diverse HIV-1 isolates
WO2022119919A1 (en) * 2020-12-01 2022-06-09 Howell Alexandra Compositions and methods for cleaving viral genomes
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
WO2023091696A1 (en) 2021-11-19 2023-05-25 Christiana Care Gene Editing Institute, Inc. Adenovirus delivery system for cancer treatment
EP4433494A2 (en) 2021-11-19 2024-09-25 Christiana Care Gene Editing Institute, Inc. Methods for intratumoral delivery of crispr/cas systems
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
US20250381213A1 (en) 2022-02-10 2025-12-18 Christiana Care Gene Editing Institute, Inc. Methods for lipid nanoparticle delivery of crispr/cas system
CN114657185B (en) * 2022-03-28 2023-11-10 福州大学 A gold magnetic nanoprobe based on ordered arrangement of aptamers and its application in field soft sponge acid detection
CN115762764A (en) * 2022-11-25 2023-03-07 中山大学附属第三医院 A prediction model and its construction method for treatment outcome of HIV-negative cryptococcal meningitis
WO2024130151A1 (en) 2022-12-16 2024-06-20 Christiana Care Gene Editing Institute, Inc. Compositions and methods for treating broad chemoresistance through chemoresistance-specific regulatory components
WO2025007140A2 (en) 2023-06-30 2025-01-02 Christiana Care Gene Editing Institute, Inc. Nras gene knockout for treatment of cancer
US20250319206A1 (en) 2024-04-04 2025-10-16 Christina Care Gene Editing Institute, Inc. Crispr/cas gene editing of neh4 and/or neh5 domains in nrf2
WO2025226816A1 (en) 2024-04-23 2025-10-30 Christiana Care Gene Editing Institute, Inc. Methods of identifying and correlating crispr-induced exon skipping to phenotypic outcomes

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
TW247876B (en) 1993-12-28 1995-05-21 New York Blood Ct Inc Pharmaceutical compositions for prevention or treating HIV-1 or HIV-2 infection
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
WO1997037005A1 (en) * 1996-04-02 1997-10-09 Progenics Pharmaceuticals, Inc. Method for preventing hiv-1 infection of cd4+ cells
US6392029B1 (en) * 1997-05-09 2002-05-21 The Research Foundation Of State University Of New York HIV chemokines
US20040132161A1 (en) * 1998-04-20 2004-07-08 Finkel Terri H. Methods and compositions for increasing CD4lymphocyte immune responsiveness
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
WO2000066759A1 (en) * 1999-04-29 2000-11-09 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6864085B2 (en) 1999-12-14 2005-03-08 Novartis Ag Bovine immunodeficiency virus (BIV) based vectors
FR2825280B1 (en) 2001-06-01 2005-04-15 Merial Sas VACCINATION AGAINST THE FELINE IMMUNODEFICIENCY VIRUS
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 (en) 2011-10-24 2013-04-24 四川百利药业有限责任公司 Preparation method of SIV (simian immunodeficiency virus) vector
GB201122458D0 (en) * 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
BR112015013784A2 (en) 2012-12-12 2017-07-11 Massachusetts Inst Technology application, manipulation and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CN113355357B (en) 2012-12-12 2024-12-03 布罗德研究所有限公司 Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
DK3553174T3 (en) 2012-12-17 2025-08-04 Harvard College RNA-GUIDED MODIFICATION OF THE HUMAN GENOME
WO2014165349A1 (en) * 2013-04-04 2014-10-09 Trustees Of Dartmouth College Compositions and methods for in vivo excision of hiv-1 proviral dna
KR102829712B1 (en) 2013-08-29 2025-07-10 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Methods and compositions for rna-guided treatment of hiv infection
AU2015266770A1 (en) * 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
CA2987927C (en) 2015-06-01 2024-03-19 Temple University - Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
JP2018527943A (en) 2015-09-28 2018-09-27 テンプル ユニバーシティー オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション Methods and compositions for the treatment of RNA-induced 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
RU2018130641A (en) 2016-01-25 2020-02-26 Эксижн Биотерапьютикс GUIDED RNA REMOVING HUMAN JC VIRUS AND OTHER POLIOMAVIRUSES
EP3407918A4 (en) 2016-01-25 2019-09-04 Excision Biotherapeutics METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
AU2017219605B2 (en) 2016-02-15 2023-04-13 Temple University - Of The Commonwealth System Of Higher Education Excision of retroviral nucleic acid sequences
WO2017176529A1 (en) 2016-04-06 2017-10-12 Temple Univesity-Of The Commonwealth System Of Higher Education Compositions for eradicating flavivirus infections in subjects
WO2017192172A1 (en) 2016-05-05 2017-11-09 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
EP3466177A4 (en) 2016-05-26 2020-01-29 Nokia Technologies Oy ADAPTATION OF CODE WORD FOR NON-ORTHOGONAL CODED ACCESS
WO2017213898A2 (en) 2016-06-07 2017-12-14 Temple University - Of The Commonwealth System Of Higher Education Rna guided compositions for preventing and treating hepatitis b virus infections
US20190085326A1 (en) 2016-09-14 2019-03-21 Temple University - Of The Commonwealth System Of Higher Education Negative feedback regulation of HIV-1 by gene editing strategy
US20190365862A1 (en) 2016-10-12 2019-12-05 Temple University - Of The Commonwealth System Of Higher Education Combination therapies for eradicating flavivirus infections in subjects
AU2018213952A1 (en) 2017-01-26 2019-08-29 Excision Biotherapeutics, Inc. Lentivirus and non-integrating lentivirus as viral vector to deliver CRISPR therapeutic
WO2018152418A1 (en) 2017-02-17 2018-08-23 Temple University - Of The Commonwealth System Of Higher Education Gene editing therapy for hiv infection via dual targeting of hiv genome and ccr5
US20200140865A1 (en) 2017-04-17 2020-05-07 Temple University - Of The Commonwealth System Of Higher Education An hiv-1 eradication strategy employing nanoformulated anti-retroviral drugs and gene editing agents
US20210060138A1 (en) 2019-03-06 2021-03-04 Temple University - Of The Commonwealth System Of Higher Education CRISPR and LASER ART Eliminates HIV

Also Published As

Publication number Publication date
CN106102781A (en) 2016-11-09
US20180221458A1 (en) 2018-08-09
US20180169195A1 (en) 2018-06-21
US20210252114A1 (en) 2021-08-19
US20160017301A1 (en) 2016-01-21
US20220313795A1 (en) 2022-10-06
MX2016002586A (en) 2016-06-14
EP3038661A4 (en) 2017-04-05
IL244220B (en) 2020-06-30
EP4385567A3 (en) 2024-08-14
US20210069303A1 (en) 2021-03-11
EP4385567A2 (en) 2024-06-19
US20180228874A1 (en) 2018-08-16
US20180207243A1 (en) 2018-07-26
JP2016534125A (en) 2016-11-04
BR112016004091A2 (en) 2017-10-17
US20240139294A1 (en) 2024-05-02
US9925248B2 (en) 2018-03-27
JP2023071897A (en) 2023-05-23
US20160250300A1 (en) 2016-09-01
SG11201601313TA (en) 2016-03-30
US20180236045A1 (en) 2018-08-23
WO2015031775A1 (en) 2015-03-05
US20180236046A1 (en) 2018-08-23
EA201600226A1 (en) 2016-09-30
IL244220A0 (en) 2016-04-21
ZA201601344B (en) 2017-05-31
EP3038661A1 (en) 2016-07-06
US20180200343A1 (en) 2018-07-19
US20180236041A1 (en) 2018-08-23
CA2922428A1 (en) 2015-03-05
US9981020B2 (en) 2018-05-29
US20230181698A1 (en) 2023-06-15
KR20230017364A (en) 2023-02-03
US20180228876A1 (en) 2018-08-16
US20180236042A1 (en) 2018-08-23
JP7118588B2 (en) 2022-08-16
US11298411B2 (en) 2022-04-12
US20180214521A1 (en) 2018-08-02
CL2016000424A1 (en) 2017-07-14
US20180303915A1 (en) 2018-10-25
US20180169194A1 (en) 2018-06-21
US11285193B2 (en) 2022-03-29
AU2020264257A1 (en) 2020-11-26
AU2014312123A1 (en) 2016-03-17
KR102829712B1 (en) 2025-07-10
AU2020264257B2 (en) 2022-10-20
US20180236044A1 (en) 2018-08-23
EA037850B1 (en) 2021-05-27
JP2020188757A (en) 2020-11-26
US20180228875A1 (en) 2018-08-16
US11291710B2 (en) 2022-04-05
US20180169193A1 (en) 2018-06-21
US20180236043A1 (en) 2018-08-23
KR20160060659A (en) 2016-05-30
EP3038661B1 (en) 2023-12-27

Similar Documents

Publication Publication Date Title
ZA201601344B (en) Methods and compositions for rna-guided treatment of hiv infection
ZA201708236B (en) Methods and compositions for rna-guided treatment of hiv infection
IL244431A0 (en) Methods and pharmaceutical compositions for the treatment of hepatitis b virus infection
PL3019503T3 (en) Polycyclic-carbamoylpyridone compounds and their use for the treatment of hiv infections
GB201320723D0 (en) Composition and methods of treatment
SI3019499T1 (en) Polycyclic-carbamoylpyridone compounds and their use for the treatment of hiv infections
IL237173A0 (en) Methods for the treatment of hepatitis b and hepatitis d infections
PT3021839T (en) Methods and compositions for treatment of fibrosis
PL3082845T3 (en) Methods and compositions for treatment of peripheral neuropathies
PL2968478T3 (en) Compositions and methods for the treatment of carboxyhemoglobinemia
IL244881B (en) Hiv treatment formulation of atazanavir and cobicistat
EP2968127A4 (en) Low-glycerin formulations for hiv treatment and prevention
AU2013902004A0 (en) Compositions for Therapy and Methods of Treatment