MX374530B - Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales. - Google Patents

Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.

Info

Publication number
MX374530B
MX374530B MX2016007324A MX2016007324A MX374530B MX 374530 B MX374530 B MX 374530B MX 2016007324 A MX2016007324 A MX 2016007324A MX 2016007324 A MX2016007324 A MX 2016007324A MX 374530 B MX374530 B MX 374530B
Authority
MX
Mexico
Prior art keywords
compositions
crispr
hbv
diseases
act
Prior art date
Application number
MX2016007324A
Other languages
English (en)
Other versions
MX2016007324A (es
Inventor
Amir Shlomai
Charles Rice
David Banjamin Turitz COX
Feng Zhang
Robert Schwartz
Sangeeta Bhatia
Vyas Ramanan
Original Assignee
Broad Inst Inc
Massachusetts Inst Technology
Univ Rockefeller
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 Broad Inst Inc, Massachusetts Inst Technology, Univ Rockefeller filed Critical Broad Inst Inc
Publication of MX2016007324A publication Critical patent/MX2016007324A/es
Publication of MX374530B publication Critical patent/MX374530B/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P31/20—Antivirals for DNA viruses
    • A61P31/22—Antivirals for DNA viruses for herpes viruses
    • 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
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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]
    • G06Q10/40—
    • H—ELECTRICITY
    • H04—ELECTRIC COMMUNICATION TECHNIQUE
    • H04L—TRANSMISSION OF DIGITAL INFORMATION, e.g. TELEGRAPHIC COMMUNICATION
    • H04L51/00—User-to-user messaging in packet-switching networks, transmitted according to store-and-forward or real-time protocols, e.g. e-mail
    • H04L51/52—User-to-user messaging in packet-switching networks, transmitted according to store-and-forward or real-time protocols, e.g. e-mail for supporting social networking services
    • H—ELECTRICITY
    • H04—ELECTRIC COMMUNICATION TECHNIQUE
    • H04L—TRANSMISSION OF DIGITAL INFORMATION, e.g. TELEGRAPHIC COMMUNICATION
    • H04L65/00—Network arrangements, protocols or services for supporting real-time applications in data packet communication
    • H04L65/1066—Session management
    • H04L65/1069—Session establishment or de-establishment
    • H—ELECTRICITY
    • H04—ELECTRIC COMMUNICATION TECHNIQUE
    • H04L—TRANSMISSION OF DIGITAL INFORMATION, e.g. TELEGRAPHIC COMMUNICATION
    • H04L67/00—Network arrangements or protocols for supporting network services or applications
    • H04L67/2866—Architectures; Arrangements
    • H04L67/30—Profiles
    • H04L67/306—User profiles
    • H—ELECTRICITY
    • H04—ELECTRIC COMMUNICATION TECHNIQUE
    • H04L—TRANSMISSION OF DIGITAL INFORMATION, e.g. TELEGRAPHIC COMMUNICATION
    • H04L67/00—Network arrangements or protocols for supporting network services or applications
    • H04L67/50—Network services
    • H04L67/53—Network services using third party service providers
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Signal Processing (AREA)
  • Computer Networks & Wireless Communication (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Business, Economics & Management (AREA)
  • General Business, Economics & Management (AREA)
  • Computing Systems (AREA)

Abstract

La invención proporciona el suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y/o actividades de secuencias diana. Se proporcionan sistemas de suministro y tejidos u órganos que se escogen como sitios diana para el suministro. También se proporcionan vectores y sistemas vectoriales, donde algunos de los cuales codifican uno o más componentes de un complejo CRISPR, así como también métodos para el diseño y uso de tales vectores. También se proporcionan métodos para dirigir la formación del complejo CRISPR en células iotas para garantizar una mejor especificidad en lo que se refiere al reconocimiento de la diana y para evitar toxicidad y para editar o modificar un sitio diana en un locus genómico de interés con el fin de alterar o mejorar el estado de una enfermedad o afección.
MX2016007324A 2013-12-12 2014-12-12 Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales. MX374530B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361915301P 2013-12-12 2013-12-12
US201462010329P 2014-06-10 2014-06-10
PCT/US2014/070135 WO2015089465A1 (en) 2013-12-12 2014-12-12 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders

Publications (2)

Publication Number Publication Date
MX2016007324A MX2016007324A (es) 2017-03-06
MX374530B true MX374530B (es) 2025-03-06

Family

ID=53371891

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016007324A MX374530B (es) 2013-12-12 2014-12-12 Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.

Country Status (13)

Country Link
US (3) US20160317677A1 (es)
EP (3) EP4183876A1 (es)
JP (1) JP2017527256A (es)
KR (1) KR20160089530A (es)
CN (2) CN111269902A (es)
AU (1) AU2014361784A1 (es)
BR (1) BR112016013207A2 (es)
CA (1) CA2932479A1 (es)
IL (1) IL246117B (es)
MX (1) MX374530B (es)
RU (1) RU2016128077A (es)
SG (1) SG10201804975PA (es)
WO (1) WO2015089465A1 (es)

Families Citing this family (232)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
SI2986729T1 (sl) 2013-04-16 2019-02-28 Regeneron Pharmaceuticals, Inc. Ciljana sprememba genoma podgane
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
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
WO2015070083A1 (en) 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
CN110951779B (zh) 2013-12-11 2024-04-16 瑞泽恩制药公司 用于靶向修饰基因组的方法和组合物
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
WO2015126927A2 (en) * 2014-02-18 2015-08-27 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
EP3498845B1 (en) * 2014-04-01 2022-06-22 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
CA2949713A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
CN107209171A (zh) * 2014-10-21 2017-09-26 普林斯顿大学理事会 用于个人样品分析的系统和方法
US20190100769A1 (en) * 2014-10-31 2019-04-04 Massachusetts Institute Of Technology Massively parallel combinatorial genetics for crispr
ES3013183T3 (en) 2014-11-21 2025-04-11 Regeneron Pharma Methods for targeted genetic modification using paired guide rnas
US11168369B2 (en) 2014-11-25 2021-11-09 The Brigham And Women's Hospital, Inc. Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease
JP7068821B2 (ja) 2014-12-03 2022-05-17 アジレント・テクノロジーズ・インク 化学修飾を有するガイドｒｎａ
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016100974A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016108926A1 (en) 2014-12-30 2016-07-07 The Broad Institute Inc. Crispr mediated in vivo modeling and genetic screening of tumor growth and metastasis
MA41349A (fr) * 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
GB2536650A (en) 2015-03-24 2016-09-28 Augmedics Ltd Method and system for combining video-based and optic-based augmented reality in a near eye display
KR102888521B1 (ko) 2015-04-06 2025-11-19 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
US10117911B2 (en) * 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
US20160346362A1 (en) * 2015-05-29 2016-12-01 Agenovir Corporation Methods and compositions for treating cytomegalovirus infections
US20180245074A1 (en) * 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
US10626393B2 (en) 2015-06-04 2020-04-21 Arbutus Biopharma Corporation Delivering CRISPR therapeutics with lipid nanoparticles
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
EP4159856A1 (en) 2015-06-18 2023-04-05 The Broad Institute, Inc. Novel crispr enzymes and systems
JP2018519811A (ja) 2015-06-29 2018-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ｃｒｉｓｐｒ ｒｎａ及び修飾単一ｃｒｉｓｐｒ ｒｎａならびにその使用
WO2017004191A1 (en) * 2015-06-30 2017-01-05 Regents Of The University Of Minnesota Transgenic mouse for expressing apobec3b
JP2017018026A (ja) * 2015-07-09 2017-01-26 国立研究開発法人医薬基盤・健康・栄養研究所 多能性幹細胞の遺伝子ターゲティング方法
EP3325018B1 (en) 2015-07-22 2025-01-15 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
AU2016299271B2 (en) 2015-07-25 2022-09-22 Valinor Aps A system, device and a method for providing a therapy or a cure for cancer and other pathological states
EP3334484B1 (en) 2015-08-12 2024-12-25 The General Hospital Corporation Compositions that promote hypoxia or the hypoxia response for the treatment and prevention of mitochondrial dysfunction
EP4177346A3 (en) 2015-08-25 2023-07-26 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
US20170087225A1 (en) * 2015-09-29 2017-03-30 Agenovir Corporation Compositions and methods for latent viral transcription regulation
CA2999922A1 (en) * 2015-09-29 2017-04-06 Agenovir Corporation Delivery methods and compositions
IL297017A (en) 2015-10-08 2022-12-01 Harvard College Multiplexed genome editing
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017070284A1 (en) * 2015-10-21 2017-04-27 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating hepatitis b virus
CA3024543A1 (en) 2015-10-22 2017-04-27 The Broad Institute, Inc. Type vi-b crispr enzymes and systems
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
CN115491373A (zh) 2015-10-30 2022-12-20 爱迪塔斯医药公司 治疗单纯疱疹病毒的crispr/cas相关方法及组合物
EP3373938A4 (en) * 2015-11-09 2019-05-15 Seattle Children's Hospital (DBA Seattle Children's Research Institute) NOVEL RNA-BASED VECTOR SYSTEM FOR TEMPORARY AND STABLE GENE EXPRESSION
US11001622B2 (en) 2015-11-19 2021-05-11 The Brigham And Women's Hospital, Inc. Method of treating autoimmune disease with lymphocyte antigen CD5-like (CD5L) protein
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
WO2017161325A1 (en) 2016-03-17 2017-09-21 Massachusetts Institute Of Technology Methods for identifying and modulating co-occurant cellular phenotypes
WO2017180915A2 (en) * 2016-04-13 2017-10-19 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017192172A1 (en) * 2016-05-05 2017-11-09 Temple University - Of The Commonwealth System Of Higher Education Rna guided eradication of varicella zoster virus
RU2745563C2 (ru) 2016-05-20 2021-03-29 Регенерон Фармасьютикалс, Инк. Способы преодоления иммунологической толерантности с использованием множества направляющих рнк
WO2017205668A1 (en) 2016-05-25 2017-11-30 Arizona Board Of Regents On Behalf Of Arizona State University Portable, low-cost pathogen detection and strain identification platform
EP3463406A4 (en) * 2016-06-01 2020-01-22 Excision Biotherapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING LYTICS AND LYSOGENIC VIRUSES
JP7436145B2 (ja) * 2016-06-03 2024-02-21 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル Ｃａｓ９ヌクレアーゼをコードする核酸の食餌コントロール発現及びその使用
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US10669558B2 (en) 2016-07-01 2020-06-02 Microsoft Technology Licensing, Llc Storage through iterative DNA editing
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
EP3494215A1 (en) 2016-08-03 2019-06-12 President and Fellows of Harvard College Adenosine nucleobase editors and uses thereof
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
KR101856345B1 (ko) * 2016-08-24 2018-06-20 경상대학교산학협력단 CRISPR/Cas9 시스템을 이용하여 APOBEC3H 및 APOBEC3CH 이중-넉아웃 고양이를 제조하는 방법
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
AU2017342543B2 (en) 2016-10-14 2024-06-27 President And Fellows Of Harvard College AAV delivery of nucleobase editors
CN117402852A (zh) * 2016-10-14 2024-01-16 精密生物科学公司 对乙肝病毒基因组中的识别序列特异性的工程化大范围核酸酶
WO2018089386A1 (en) 2016-11-11 2018-05-17 The Broad Institute, Inc. Modulation of intestinal epithelial cell differentiation, maintenance and/or function through t cell action
WO2018091971A1 (en) 2016-11-15 2018-05-24 Genomic Vision Method for the monitoring of modified nucleases induced-gene editing events by molecular combing
CN106729753A (zh) * 2016-12-16 2017-05-31 谭旭 抗乙型肝炎病毒的递送系统和生物制剂
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
KR101796036B1 (ko) * 2016-12-29 2017-11-10 주식회사 무진메디 Cas9 단백질, KRAS 유전자의 발현을 억제하는 가이드 RNA 및 양이온성 폴리머의 복합체가 봉입된 나노 리포좀 전달체 조성물 또는 이를 함유하는 KRAS 유전자 변이에 따른 항암제 저항성 대장암 치료제
CN108342387B (zh) * 2017-01-24 2021-09-24 谭旭 Pcsk9抑制剂类降血脂药的递送系统和生物制剂
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
WO2018175924A1 (en) 2017-03-24 2018-09-27 The Broad Institute, Inc. Methods and compositions for regulating innate lymphoid cell inflammatory responses
WO2018183908A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating ovarian tumors
WO2018191553A1 (en) 2017-04-12 2018-10-18 Massachusetts Eye And Ear Infirmary Tumor signature for metastasis, compositions of matter methods of use thereof
US12331319B2 (en) 2017-04-12 2025-06-17 The Broad Institute, Inc. Respiratory and sweat gland ionocytes
US12058986B2 (en) 2017-04-20 2024-08-13 Egenesis, Inc. Method for generating a genetically modified pig with inactivated porcine endogenous retrovirus (PERV) elements
EP3612232A1 (en) 2017-04-21 2020-02-26 The Broad Institute, Inc. Targeted delivery to beta cells
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
WO2018209324A2 (en) 2017-05-11 2018-11-15 The Broad Institute, Inc. Methods and compositions of use of cd8+ tumor infiltrating lymphocyte subtypes and gene signatures thereof
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
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US20220090127A1 (en) * 2017-06-02 2022-03-24 Institut National De La Sante Et De La Recherche Medicale (Inserm Viral vector combining gene therapy and genome editing approaches for gene therapy of genetic disorders
WO2018226762A1 (en) 2017-06-05 2018-12-13 Fred Hutchinson Cancer Research Center Genomic safe harbors for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies
WO2018232195A1 (en) 2017-06-14 2018-12-20 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
WO2018234239A1 (en) * 2017-06-19 2018-12-27 Cellectis Anti-hbv combination therapies involving specific endonucleases
WO2018237369A2 (en) * 2017-06-23 2018-12-27 Vical Incorporated Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection
WO2019005884A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION
WO2019014581A1 (en) 2017-07-14 2019-01-17 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODULATING THE ACTIVITY OF A CYTOTOXIC LYMPHOCYTE
US12105089B2 (en) 2017-07-17 2024-10-01 The Broad Institute, Inc. Cell atlas of the healthy and ulcerative colitis human colon
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019027871A1 (en) * 2017-07-31 2019-02-07 Ingateygen Llc CACAHUETES WITH REDUCED ALLERGEN LEVELS
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
AU2018336128B2 (en) 2017-09-19 2023-01-05 Tropic Biosciences UK Limited Modifying the specificity of plant non-coding RNA molecules for silencing gene expression
CN107760714A (zh) * 2017-09-20 2018-03-06 青岛海洋生物医药研究院股份有限公司 转基因构建物及其应用
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
SG11202002130WA (en) * 2017-09-28 2020-04-29 Toolgen Inc Artificial genome manipulation for gene expression regulation
US11572574B2 (en) 2017-09-28 2023-02-07 Toolgen Incorporated Artificial genome manipulation for gene expression regulation
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US11905513B2 (en) * 2017-10-13 2024-02-20 Mbp Titan, Llc Advanced genome editing
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
WO2019079362A1 (en) 2017-10-16 2019-04-25 Massachusetts Institute Of Technology HOST-PATHOGENIC INTERACTION OF MYCOBACTERIUM TUBERCULOSIS
CA3079681A1 (en) 2017-10-20 2019-04-25 Fred Hutchinson Cancer Research Center Systems and methods to produce b cells genetically modified to express selected antibodies
US12221720B2 (en) 2017-11-13 2025-02-11 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
WO2019094955A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for targeting developmental and oncogenic programs in h3k27m gliomas
CN120310773A (zh) * 2017-11-16 2025-07-15 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
US12521201B2 (en) 2017-12-07 2026-01-13 Augmedics Ltd. Spinous process clamp
US12458411B2 (en) 2017-12-07 2025-11-04 Augmedics Ltd. Spinous process clamp
WO2019113506A1 (en) 2017-12-07 2019-06-13 The Broad Institute, Inc. Methods and compositions for multiplexing single cell and single nuclei sequencing
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN111492059A (zh) * 2017-12-20 2020-08-04 帝斯曼知识产权资产管理有限公司 在宿主细胞中进行基因组编辑的方法
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US11994512B2 (en) 2018-01-04 2024-05-28 Massachusetts Institute Of Technology Single-cell genomic methods to generate ex vivo cell systems that recapitulate in vivo biology with improved fidelity
US11795456B2 (en) * 2018-04-16 2023-10-24 Georgia Tech Research Corporation MRNA driven expression of RNA editors for treatment of pathologies
US12338486B2 (en) 2018-04-19 2025-06-24 Massachusetts Institute Of Technology Single-stranded break detection in double-stranded DNA
HUE059223T2 (hu) 2018-04-24 2022-10-28 Kws Saat Se & Co Kgaa Jobb emészthetõségû növények és marker haplotípusok
US11957695B2 (en) 2018-04-26 2024-04-16 The Broad Institute, Inc. Methods and compositions targeting glucocorticoid signaling for modulating immune responses
WO2019210268A2 (en) 2018-04-27 2019-10-31 The Broad Institute, Inc. Sequencing-based proteomics
WO2019211741A1 (en) 2018-05-02 2019-11-07 Augmedics Ltd. Registration of a fiducial marker for an augmented reality system
WO2019213660A2 (en) 2018-05-04 2019-11-07 The Broad Institute, Inc. Compositions and methods for modulating cgrp signaling to regulate innate lymphoid cell inflammatory responses
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019232542A2 (en) 2018-06-01 2019-12-05 Massachusetts Institute Of Technology Methods and compositions for detecting and modulating microenvironment gene signatures from the csf of metastasis patients
EP3578658A1 (en) * 2018-06-08 2019-12-11 Johann Wolfgang Goethe-Universität Frankfurt Method for generating a gene editing vector with fixed guide rna pairs
US12036240B2 (en) 2018-06-14 2024-07-16 The Broad Institute, Inc. Compositions and methods targeting complement component 3 for inhibiting tumor growth
RU2703532C1 (ru) * 2018-06-15 2019-10-21 ФБУН Центральный НИИ эпидемиологии Роспотребнадзора Система для активации цитидиндезаминаз APOBEC/AID человека и/или урацил-ДНК-гликозилазы UNG человека и ее применение для элиминации ккз ДНК вируса гепатита B из клеток человека, в частности из гепатоцитов
CN110616233B (zh) * 2018-06-20 2022-02-22 西安桑尼赛尔生物医药有限公司 CRISPR-Cas9高效敲除原代T细胞基因的方法及其应用
JP2021528975A (ja) 2018-06-26 2021-10-28 ザ・ブロード・インスティテュート・インコーポレイテッド Ｃｒｉｓｐｒ／ｃａｓ及びトランスポザーゼを利用した増幅用の組成物、システム、及び方法
EP3814527B1 (en) 2018-06-26 2023-02-22 Massachusetts Institute of Technology Crispr effector system based amplification methods, systems, and diagnostics
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
KR20210056329A (ko) 2018-08-07 2021-05-18 더 브로드 인스티튜트, 인코퍼레이티드 신규 cas12b 효소 및 시스템
US20210324357A1 (en) 2018-08-20 2021-10-21 The Brigham And Women's Hospital, Inc. Degradation domain modifications for spatio-temporal control of rna-guided nucleases
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
AU2019329686A1 (en) 2018-08-27 2020-12-03 Regeneron Pharmaceuticals, Inc. Use of raman spectroscopy in downstream purification
CN109321596B (zh) * 2018-09-05 2021-12-17 暨南大学 一种包载蛋白的外泌体的制备方法与应用
EP3847650A1 (en) 2018-09-06 2021-07-14 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP3849565A4 (en) 2018-09-12 2022-12-28 Fred Hutchinson Cancer Research Center REDUCTION OF CD33 EXPRESSION TO SELECTIVELY PROTECT THERAPEUTIC CELLS
KR20240093924A (ko) 2018-09-18 2024-06-24 브이엔브이 뉴코 인크. Arc 기반 캡시드 및 이의 용도
US12312584B2 (en) 2018-10-02 2025-05-27 Exosome Therapeutics, Inc. cGMP exosome loaded therapeutics for treating sickle cell disease
JP2020055774A (ja) * 2018-10-02 2020-04-09 国立大学法人 鹿児島大学 抗ウイルス薬
WO2020077178A1 (en) * 2018-10-12 2020-04-16 Ann & Robert H. Lurie Children's Hospital of Chicago Plga-peg/pei nanoparticles and methods of use
WO2020077236A1 (en) 2018-10-12 2020-04-16 The Broad Institute, Inc. Method for extracting nuclei or whole cells from formalin-fixed paraffin-embedded tissues
WO2020081730A2 (en) 2018-10-16 2020-04-23 Massachusetts Institute Of Technology Methods and compositions for modulating microenvironment
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12165743B2 (en) 2018-11-09 2024-12-10 The Broad Institute, Inc. Compressed sensing for screening and tissue imaging
US12402610B2 (en) 2018-11-09 2025-09-02 The Broad Institute, Inc. Methods and compositions for modulating innate lymphoid cell pathogenic effectors
WO2020106771A1 (en) * 2018-11-19 2020-05-28 Exosome Therapeutics, Inc. Compositions and methods for producing exosome loaded therapeutics for the treatment of multiple oncological disorders
US11766296B2 (en) 2018-11-26 2023-09-26 Augmedics Ltd. Tracking system for image-guided surgery
CA3121191A1 (en) 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
MX2021006977A (es) * 2018-12-12 2021-10-13 Flagship Pioneering Innovations V Inc Anellosomas y metodos de uso.
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
US11739156B2 (en) 2019-01-06 2023-08-29 The Broad Institute, Inc. Massachusetts Institute of Technology Methods and compositions for overcoming immunosuppression
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3930694A1 (en) * 2019-02-28 2022-01-05 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
AU2020242032A1 (en) 2019-03-19 2021-10-07 Massachusetts Institute Of Technology Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20200123392A (ko) * 2019-04-18 2020-10-29 주식회사 툴젠 B형 간염 바이러스의 증식을 억제하는 조성물 및 이의 방법
EP3965832A4 (en) * 2019-05-10 2024-02-07 Beam Therapeutics, Inc. Compositions and methods for treating hepatitis b
UY38693A (es) 2019-05-13 2020-12-31 Kws Saat Se & Co Kgaa Tolerancia a la sequía en maíz
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
AR118995A1 (es) 2019-05-25 2021-11-17 Kws Saat Se & Co Kgaa Mejorador de la inducción de haploides
WO2020243661A1 (en) 2019-05-31 2020-12-03 The Broad Institute, Inc. Methods for treating metabolic disorders by targeting adcy5
US20220257796A1 (en) 2019-07-02 2022-08-18 Fred Hutchinson Cancer Research Center Recombinant ad35 vectors and related gene therapy improvements
US12178666B2 (en) 2019-07-29 2024-12-31 Augmedics Ltd. Fiducial marker
US11980506B2 (en) 2019-07-29 2024-05-14 Augmedics Ltd. Fiducial marker
EP3772542A1 (en) 2019-08-07 2021-02-10 KWS SAAT SE & Co. KGaA Modifying genetic variation in crops by modulating the pachytene checkpoint protein 2
WO2021030642A1 (en) 2019-08-14 2021-02-18 Memorial Sloan Kettering Cancer Center Methods of treating p53 mutant cancers using ogdh inhibitors cross-reference to related applications
WO2021030735A1 (en) * 2019-08-15 2021-02-18 The Rockefeller University Crispr genome editing with cell surface display to produce homozygously edited eukaryotic cells
WO2021034373A1 (en) * 2019-08-19 2021-02-25 Minghong Zhong Conjugates of guide rna-cas protein complex
US20250381301A1 (en) * 2019-08-19 2025-12-18 Minghong Zhong Conjugates of Guide RNA-Cas Protein Complex
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
US20220315645A1 (en) * 2019-09-11 2022-10-06 The Rockefeller University Compositions and methods related to human neutralizing antibodies to hepatitis b
US11981922B2 (en) 2019-10-03 2024-05-14 Dana-Farber Cancer Institute, Inc. Methods and compositions for the modulation of cell interactions and signaling in the tumor microenvironment
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021074367A1 (en) 2019-10-17 2021-04-22 KWS SAAT SE & Co. KGaA Enhanced disease resistance of crops by downregulation of repressor genes
US11382712B2 (en) 2019-12-22 2022-07-12 Augmedics Ltd. Mirroring in image guided surgery
EP3872190A1 (en) 2020-02-26 2021-09-01 Antibodies-Online GmbH A method of using cut&run or cut&tag to validate crispr-cas targeting
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
PE20230080A1 (es) 2020-05-29 2023-01-11 Kws Saat Se And Co Kgaa Induccion de haploides en plantas
US11389252B2 (en) 2020-06-15 2022-07-19 Augmedics Ltd. Rotating marker for image guided surgery
US12502163B2 (en) 2020-09-09 2025-12-23 Augmedics Ltd. Universal tool adapter for image-guided surgery
US12239385B2 (en) 2020-09-09 2025-03-04 Augmedics Ltd. Universal tool adapter
EP4001429A1 (en) 2020-11-16 2022-05-25 Antibodies-Online GmbH Analysis of crispr-cas binding and cleavage sites followed by high-throughput sequencing (abc-seq)
NL2027654B1 (en) * 2021-02-08 2022-09-09 Nutcracker Therapeutics Inc Methods for manufacturing a syntetic template
CN113249384A (zh) * 2021-04-27 2021-08-13 重庆医科大学 可靶向编辑HBV cccDNA的特异sgRNA序列及其应用
CN115494031B (zh) * 2021-06-18 2024-06-18 北京大学 一种基于CRISPR/dCas9系统与寡核苷酸探针的活细胞DNA标记信号放大方法
US11896445B2 (en) 2021-07-07 2024-02-13 Augmedics Ltd. Iliac pin and adapter
WO2023283622A1 (en) 2021-07-08 2023-01-12 Montana State University Crispr-based programmable rna editing
US11814689B2 (en) 2021-07-21 2023-11-14 Montana State University Nucleic acid detection using type III CRISPR complex
CA3226445A1 (en) * 2021-07-22 2023-01-26 Rafi EMMANUEL Hepatitis b virus (hbv) knockouts
US12150821B2 (en) 2021-07-29 2024-11-26 Augmedics Ltd. Rotating marker and adapter for image-guided surgery
CN118368974A (zh) 2021-07-30 2024-07-19 科沃施种子欧洲股份两合公司 具有提高的消化率和标记单倍型的植物
WO2023021450A1 (en) 2021-08-18 2023-02-23 Augmedics Ltd. Stereoscopic display and digital loupe for augmented-reality near-eye display
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
US20250257340A1 (en) * 2022-02-23 2025-08-14 Excision Biotherapeutics Inc Guide nucleic acid identification and methods of use
CN120944860A (zh) 2022-03-08 2025-11-14 中国科学院遗传与发育生物学研究所 胞嘧啶脱氨酶及其在碱基编辑中的用途
WO2023203521A1 (en) 2022-04-21 2023-10-26 Augmedics Ltd. Systems and methods for medical image visualization
WO2023232109A1 (zh) 2022-06-01 2023-12-07 中国科学院遗传与发育生物学研究所 新的crispr基因编辑系统
AU2023289696A1 (en) 2022-06-24 2025-01-16 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
KR20250077608A (ko) * 2022-08-19 2025-05-30 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통한 b형 간염 바이러스 조절용 조성물, 시스템 및 방법
KR20240026435A (ko) * 2022-08-19 2024-02-28 서울대학교산학협력단 엑소좀을 이용한 생식세포 내 물질 전달 및 유전자 편집 시스템
US20250386788A1 (en) 2022-08-26 2025-12-25 KWS SAAT SE & Co. KGaA Use of paired genes in hybrid breeding
EP4587881A1 (en) 2022-09-13 2025-07-23 Augmedics Ltd. Augmented reality eyewear for image-guided medical intervention
WO2024186890A1 (en) * 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024236547A1 (en) 2023-05-18 2024-11-21 Inceptor Bio, Llc Modified phagocytic cells expressing chimeric antigen receptors comprising a herpes virus entry mediator (hvem) co-stimulatory domain and uses thereof
TW202503061A (zh) * 2023-06-16 2025-01-16 大陸商益杰立科（上海）生物科技有限公司 靶向ｂ型肝炎病毒基因的表觀編輯工具
WO2025046513A1 (en) 2023-08-29 2025-03-06 Inceptor Bio, Llc Methods of manufacturing myeloid-derived cells from hematopoietic stem cells and compositions and uses thereof
CN116970609B (zh) * 2023-09-05 2024-08-27 深圳市艾迪贝克生物医药有限公司 用于清除hbv病毒的基因片段、其工具系统及应用
CN119570025A (zh) * 2024-11-19 2025-03-07 中国药科大学 一种聚乙烯亚胺衍生物及其作为基因药物载体应用

Family Cites Families (225)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US101A (en) 1836-12-06 Method of jcakibtg and furling iw sails fob ships
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
EP0264166B1 (en) 1986-04-09 1996-08-21 Genzyme Corporation Transgenic animals secreting desired proteins into milk
JPS6395765U (es) 1986-12-11 1988-06-21
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US4873316A (en) 1987-06-23 1989-10-10 Biogen, Inc. Isolation of exogenous recombinant proteins from the milk of transgenic mammals
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5122457A (en) 1989-10-19 1992-06-16 Schering Corporation Expression systems utilizing bacteriophage t7 promoters, gene sequences, and t7 rna polymerase
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US7150982B2 (en) 1991-09-09 2006-12-19 Third Wave Technologies, Inc. RNA detection assays
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
CN1127527A (zh) 1993-05-17 1996-07-24 加利福尼亚大学董事会 人免疫缺陷型病毒感染和艾滋病的核酶基因治疗方法
US5385834A (en) 1993-08-13 1995-01-31 Georgia Tech Research Corporation Mutant T7 RNA polymerase GP1(lys222) exhibiting altered promoter recognition
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
AU698739B2 (en) 1995-06-06 1998-11-05 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5985662A (en) 1995-07-13 1999-11-16 Isis Pharmaceuticals Inc. Antisense inhibition of hepatitis B virus replication
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
GB9907461D0 (en) 1999-03-31 1999-05-26 King S College London Neurite regeneration
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
NZ520579A (en) 1997-10-24 2004-08-27 Invitrogen Corp Recombinational cloning using nucleic acids having recombination sites and methods for synthesizing double stranded nucleic acids
US6750059B1 (en) 1998-07-16 2004-06-15 Whatman, Inc. Archiving of vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6479808B1 (en) 1999-07-07 2002-11-12 The United States Of America As Represented By The Administrator Of The National Aeronautics And Space Administration Method and systems for collecting data from multiple fields of view
GB0024550D0 (es) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
WO2002074968A1 (en) 2001-03-16 2002-09-26 Naoya Kobayashi Method for proliferating a liver cell, a liver cell obtained thereby, and use thereof
AU2002307145B2 (en) 2001-04-05 2007-11-29 The John Hopkins University Chimeric vaccines
WO2003006477A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts IN VIVO PRODUCTION OF SMALL INTERFERING RNAs THAT MEDIATE GENE SILENCING
EP1572885A2 (en) 2001-08-08 2005-09-14 Genzyme Corporation Methods for treating diabetes and other blood sugar disorders
WO2003016338A1 (en) 2001-08-15 2003-02-27 Parker Hughes Institute Crystal structure of the btk kinase domain
AU2002336760A1 (en) 2001-09-26 2003-06-10 Mayo Foundation For Medical Education And Research Mutable vaccines
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
WO2003056022A2 (en) 2001-12-21 2003-07-10 Oxford Biomedica (Uk) Limited Method for producing a transgenic organism using a lentiviral expression vector such as eiav
WO2003087993A2 (en) 2002-04-09 2003-10-23 Beattie Kenneth L Oligonucleotide probes for genosensor chips
JP2006513694A (ja) 2002-06-11 2006-04-27 ザ スクリップス リサーチ インスティテュート 人工転写因子
WO2004002453A1 (en) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL BASED RNA INTERFERENCE AND ASSOCIATED METHODS AND COMPOSITIONS
AU2003290937A1 (en) 2002-11-15 2004-06-15 Trustees Of Boston University Cis/trans riboregulators
US20060178297A1 (en) 2003-01-28 2006-08-10 Troy Carol M Systems and methods for silencing expression of a gene in a cell and uses thereof
JP4980716B2 (ja) 2003-06-12 2012-07-18 アルナイラム ファーマシューティカルズ， インコーポレイテッド 遺伝子サイレンシングに有用な保存されたｈｂｖ及びｈｃｖ配列
EP1644534A4 (en) 2003-07-03 2006-08-09 Univ California GENOMKARTING OF FUNCTIONAL DNA ELEMENTS AND CELLULAR PROTEINS
US20050064595A1 (en) 2003-07-16 2005-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
WO2005014791A2 (en) 2003-08-08 2005-02-17 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2551022C (en) 2003-09-15 2013-06-04 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
GB0325379D0 (en) 2003-10-30 2003-12-03 Oxford Biomedica Ltd Vectors
FR2862659B1 (fr) 2003-11-21 2006-02-10 Pasteur Institut Genome de legionella pneumophila souche paris- applications diagnostiques et epidemiologiques
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US20070244031A1 (en) 2004-01-27 2007-10-18 Quan Lu Methods and Compositions for Homozygous Gene Inactivation Using Collections of Pre-Defined Nucleotide Sequences Complementary Chromosomal Transcripts
US20050220796A1 (en) 2004-03-31 2005-10-06 Dynan William S Compositions and methods for modulating DNA repair
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1766035B1 (en) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
EP2397490B1 (en) 2004-07-16 2013-09-04 THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Vaccine constructs and combinations of vaccines designed to improve the breadth of the immune response to diverse strains and clades of HIV
GB0422877D0 (en) 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006116756A1 (en) 2005-04-28 2006-11-02 Benitec, Limited. Multiple-rnai expression cassettes for simultaneous delivery of rnai agents related to heterozygotic expression patterns
US7892224B2 (en) 2005-06-01 2011-02-22 Brainlab Ag Inverse catheter planning
CA2615532C (en) 2005-07-26 2016-06-28 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
CA2961849C (en) 2005-08-26 2022-08-02 Dupont Nutrition Biosciences Aps Use of crispr associated genes (cas)
WO2007048046A2 (en) 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US8362229B2 (en) 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
EP1994182B1 (en) 2006-03-15 2019-05-29 Siemens Healthcare Diagnostics Inc. Degenerate nucleobase analogs
EP2584047B1 (en) 2006-05-11 2014-11-19 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. Method for delivery across the blood brain barrier
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
WO2008093152A1 (en) 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
US8173792B2 (en) 2007-02-09 2012-05-08 The Board Of Trustees Of The Leland Stanford Junior University Method for regulating protein function in cells using synthetic small molecules
TR201905633T4 (tr) 2007-03-02 2019-05-21 Dupont Nutrition Biosci Aps İyileştirilmiş faj direnci olan kültürler.
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
WO2008149176A1 (en) 2007-06-06 2008-12-11 Cellectis Meganuclease variants cleaving a dna target sequence from the mouse rosa26 locus and uses thereof
CN103083794B (zh) 2007-08-14 2016-03-02 弗雷德哈钦森癌症研究中心 用于递送治疗药物的针阵列组件和方法
EP2225002A4 (en) 2007-12-31 2011-06-22 Nanocor Therapeutics Inc RNA INTERFERENCE FOR THE TREATMENT OF CARDIAC SUFFICIENCIES
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
HUE034483T2 (en) 2008-04-15 2018-02-28 Protiva Biotherapeutics Inc New lipid preparations for introducing a nucleic acid
WO2010001325A2 (en) 2008-06-30 2010-01-07 Silenseed Ltd Methods, compositions and systems for local delivery of drugs
US20110117189A1 (en) 2008-07-08 2011-05-19 S.I.F.I. Societa' Industria Farmaceutica Italiana S.P.A. Ophthalmic compositions for treating pathologies of the posterior segment of the eye
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
IL302142B2 (en) 2008-10-20 2024-07-01 Alnylam Pharmaceuticals Inc Compounds and methods for inhibiting transthyretin expression
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
JP5681114B2 (ja) 2008-12-04 2015-03-04 サンガモ バイオサイエンシーズ， インコーポレイテッド 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010075424A2 (en) 2008-12-22 2010-07-01 The Regents Of University Of California Compositions and methods for downregulating prokaryotic genes
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
HRP20150564T1 (hr) 2009-04-07 2015-08-28 Dow Agrosciences Llc Nanoäśesticama posredovana sekvenca specifiäśnih nukleaza
US20120164118A1 (en) 2009-05-04 2012-06-28 Fred Hutchinson Cancer Research Center Cocal vesiculovirus envelope pseudotyped retroviral vectors
WO2011036510A1 (en) * 2009-09-24 2011-03-31 Cellectis Meganuclease variants cleaving the genome of the herpes simplex virus and uses thereof
KR20100133319A (ko) 2009-06-11 2010-12-21 주식회사 툴젠 표적 특이적인 게놈의 재배열을 위한 표적 특이적 뉴클레아제 및 이의 용도
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011000108A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
JP2013500018A (ja) 2009-07-24 2013-01-07 シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニー ゲノム編集のための方法
EP2461819A4 (en) * 2009-07-28 2013-07-31 Sangamo Biosciences Inc METHODS AND COMPOSITIONS FOR TREATING TRI-NUCLEOTIDE REPEAT DISORDERS
WO2011028929A2 (en) 2009-09-03 2011-03-10 The Regents Of The University Of California Nitrate-responsive promoter
US8889394B2 (en) 2009-09-07 2014-11-18 Empire Technology Development Llc Multiple domain proteins
CA2782014C (en) 2009-11-27 2021-08-31 Basf Plant Science Company Gmbh Optimized endonucleases and uses thereof
DK2816112T3 (en) 2009-12-10 2018-11-19 Univ Minnesota TAL effector-mediated DNA modification
AU2010334799A1 (en) 2009-12-23 2012-07-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Influenza targets
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
CA2796600C (en) 2010-04-26 2019-08-13 Sangamo Biosciences, Inc. Genome editing of a rosa locus using zinc-finger nucleases
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
ES2590343T3 (es) 2010-05-10 2016-11-21 The Regents Of The University Of California Composiciones de endorribonucleasas y métodos de uso de las mismas
EP3156062A1 (en) 2010-05-17 2017-04-19 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
JP5965392B2 (ja) 2010-05-28 2016-08-03 オックスフォード バイオメディカ （ユーケー） リミテッド 脳へのレンチウイルスベクターの送達
KR101971741B1 (ko) 2010-07-23 2019-08-14 시그마-알드리치 컴퍼니., 엘엘씨 표적화 엔도뉴클레아제 및 단일-가닥 핵산을 사용하는 게놈 편집
WO2012018697A1 (en) 2010-08-02 2012-02-09 Integrated Dna Technologies, Inc. Methods for predicting stability and melting temperatures of nucleic acid duplexes
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
ES2562421T3 (es) 2010-10-12 2016-03-04 The Children's Hospital Of Philadelphia Métodos y composiciones para tratar la hemofilia B
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
EP2699270B1 (en) 2011-04-22 2017-06-21 The Regents of The University of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA2834375C (en) 2011-04-27 2020-07-14 Amyris, Inc. Methods for genomic modification
US20120295960A1 (en) 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
AU2012318562A1 (en) 2011-10-06 2014-04-10 Sangamo Therapeutics, Inc. Methods and compositions for regulating HIV infection
EP2591770B1 (en) 2011-11-14 2016-03-16 Silenseed Ltd Compositions for siRNA delivery and methods of manufacturing and using same
EP2780376B1 (en) 2011-11-18 2017-03-08 Université Laval Methods and products for increasing frataxin levels and uses thereof
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
US20130156849A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
AU2013204327B2 (en) 2012-04-20 2016-09-01 Aviagen Cell transfection method
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
AU2013267209B2 (en) 2012-06-01 2017-02-02 Baruch S. Blumberg Institute Modulation of hepatitis B virus cccDNA transcription
EP3494997B1 (en) 2012-07-25 2019-09-18 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
JP5730249B2 (ja) 2012-08-07 2015-06-03 株式会社ミヤコシ ラベル原紙の粕上げ方法及び粕上げ装置
EP2695818A1 (en) 2012-08-10 2014-02-12 Marel Limited A labeling device for labeling objects, in particular moving objects
JP5632885B2 (ja) 2012-08-31 2014-11-26 株式会社日本自動車部品総合研究所 電力変換装置
CA2886684C (en) 2012-10-10 2023-09-19 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
CN110066775B (zh) 2012-10-23 2024-03-19 基因工具股份有限公司 用于切割靶dna的组合物及其用途
ES2757325T3 (es) 2012-12-06 2020-04-28 Sigma Aldrich Co Llc Modificación y regulación del genoma en base a CRISPR
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
EP2932421A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Methods, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
EP3825401A1 (en) 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
IL300461A (en) 2012-12-12 2023-04-01 Harvard College Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
CN113528577B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
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
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
US11135273B2 (en) 2013-02-07 2021-10-05 The Rockefeller University Sequence specific antimicrobials
US9163837B2 (en) 2013-02-27 2015-10-20 Siemens Aktiengesellschaft Flow conditioner in a combustor of a gas turbine engine
US9260752B1 (en) 2013-03-14 2016-02-16 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
CN110540991B (zh) 2013-03-15 2023-10-24 通用医疗公司 使用截短的引导RNA(tru-gRNA)提高RNA引导的基因组编辑的特异性
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
KR102109069B1 (ko) 2013-03-27 2020-05-12 윌코아게 디바이스들을 인라인 검사 및/또는 테스트하는 방법 및 그와 같은 방법을 수행하는 장치
EP4286517A3 (en) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6576904B2 (ja) * 2013-04-04 2019-09-18 トラスティーズ・オブ・ダートマス・カレッジ ＨＩＶ−１プロウイルスＤＮＡのｉｎｖｉｖｏ切除のための組成物及び方法
EP3730615A3 (en) 2013-05-15 2020-12-09 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2913865C (en) 2013-05-29 2022-07-19 Cellectis A method for producing precise dna cleavage using cas9 nickase activity
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
CA3176690A1 (en) 2013-06-04 2014-12-11 President And Fellows Of Harvard College Rna-guided transcriptional regulation
WO2014197748A2 (en) 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
KR20160044457A (ko) 2013-06-17 2016-04-25 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작을 위한 탠덤 안내 시스템, 방법 및 조성물의 전달, 조작 및 최적화
CN105683379A (zh) 2013-06-17 2016-06-15 布罗德研究所有限公司 用于对有丝分裂后细胞的疾病和障碍进行靶向和建模的系统、方法和组合物的递送、工程化和优化
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
AU2014281031B2 (en) 2013-06-17 2020-05-21 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components
KR20160034901A (ko) 2013-06-17 2016-03-30 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작에 최적화된 ｃｒｉｓｐｒ-ｃａｓ 이중 닉카아제 시스템, 방법 및 조성물
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
CA2917348A1 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
CN103388006B (zh) 2013-07-26 2015-10-28 华东师范大学 一种基因定点突变的构建方法
CA2922428A1 (en) * 2013-08-29 2015-03-05 Temple University Of The Commonwealth System Of Higher Education Methods and compositions for rna-guided treatment of hiv infection
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
WO2015065964A1 (en) 2013-10-28 2015-05-07 The Broad Institute Inc. Functional genomics using crispr-cas systems, compositions, methods, screens and applications thereof
WO2015070083A1 (en) 2013-11-07 2015-05-14 Editas Medicine,Inc. CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
CA2930877A1 (en) 2013-11-18 2015-05-21 Crispr Therapeutics Ag Crispr-cas system materials and methods
CN105899657A (zh) 2013-12-12 2016-08-24 布罗德研究所有限公司 用于改变基因产物表达的crispr-cas系统和方法、结构信息以及诱导型模块化cas酶
AU2014362245A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
BR112016013213A2 (pt) 2013-12-12 2017-12-05 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas
CN103668472B (zh) 2013-12-31 2014-12-24 北京大学 利用CRISPR/Cas9系统构建真核基因敲除文库的方法
WO2015113063A1 (en) 2014-01-27 2015-07-30 Georgia Tech Research Corporation Methods and systems for identifying crispr/cas off-target sites
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
JP6323228B2 (ja) 2014-07-18 2018-05-16 富士電機株式会社 電力変換装置
US9932566B2 (en) 2014-08-07 2018-04-03 Agilent Technologies, Inc. CIS-blocked guide RNA
ES2780904T3 (es) 2014-08-17 2020-08-27 Broad Inst Inc Edición genómica usando nickasas Cas9
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
CA2978314A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
CN114099712A (zh) 2015-05-06 2022-03-01 斯尼普技术有限公司 改变微生物种群和改善微生物群
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
CN108290933A (zh) 2015-06-18 2018-07-17 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
JP6186470B2 (ja) 2016-04-20 2017-08-23 パイオニア株式会社 音響装置、音量制御方法、音量制御プログラム及び記録媒体
EP3585898A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
CN107939288B (zh) 2017-11-14 2019-04-02 中国科学院地质与地球物理研究所 一种非旋转套的防转装置以及旋转导向装置
CA3123886A1 (en) 2019-01-17 2020-07-23 James DAHLMAN Drug delivery systems containing oxidized cholesterols
EP4003967A4 (en) 2019-07-29 2024-01-17 Georgia Tech Research Corporation NANOMATERIALS WITH RESTRICTED LIPIDS AND THEIR USES

Also Published As

Publication number Publication date
US20200389425A1 (en) 2020-12-10
IL246117B (en) 2020-03-31
CN105899658B (zh) 2020-02-18
US20160317677A1 (en) 2016-11-03
RU2016128077A3 (es) 2018-12-06
JP2017527256A (ja) 2017-09-21
MX2016007324A (es) 2017-03-06
EP4183876A1 (en) 2023-05-24
US12251450B2 (en) 2025-03-18
IL246117A0 (en) 2016-07-31
WO2015089465A1 (en) 2015-06-18
BR112016013207A2 (pt) 2017-09-26
EP3540051A1 (en) 2019-09-18
SG10201804975PA (en) 2018-07-30
CA2932479A1 (en) 2015-06-18
CN105899658A (zh) 2016-08-24
EP3540051B1 (en) 2022-08-17
EP3080261A1 (en) 2016-10-19
US20250381297A1 (en) 2025-12-18
KR20160089530A (ko) 2016-07-27
RU2016128077A (ru) 2018-12-06
CN111269902A (zh) 2020-06-12
EP3080261B1 (en) 2019-05-22
AU2014361784A1 (en) 2016-06-23

Similar Documents

Publication Publication Date Title
MX374530B (es) Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.
MX374532B (es) Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
MX374529B (es) Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
MX374531B (es) Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas.
MX389793B (es) Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas.
MX2021001308A (es) Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificacion dirigida y terapia hepaticas.
SG10201804973TA (en) Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
MX2016007327A (es) Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para dirigirlos a trastornos y enfermedades usando componentes para suministro de particulas.
DOP2018000227A (es) Inhibidores de bromodominios
WO2015175642A3 (en) Methods and compositions for prevention or treatment of a disease
EP4245853A3 (en) Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
WO2014204724A8 (en) Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
MX392026B (es) Compuestos heterocíclicos como inmunomoduladores.
DOP2016000298A (es) Pirazolopiridinas y pirazolopirimidinas
MX388693B (es) Composiciones de nanoparticulas para terapia sostenida.
CR20190156A (es) Derivados de bipirazol como inhibidores jak
PE20160930A1 (es) Nanoparticulas polimericas terapeuticas y metodos para su elaboracion y uso.

Legal Events

Date Code Title Description
FG Grant or registration