ATE299189T1 - Retrovirale vektoren, die funktionelle spleiss- donor- und spleiss-akzeptor-stellen enthalten - Google Patents

Retrovirale vektoren, die funktionelle spleiss- donor- und spleiss-akzeptor-stellen enthalten

Info

Publication number
ATE299189T1
ATE299189T1 AT98944085T AT98944085T ATE299189T1 AT E299189 T1 ATE299189 T1 AT E299189T1 AT 98944085 T AT98944085 T AT 98944085T AT 98944085 T AT98944085 T AT 98944085T AT E299189 T1 ATE299189 T1 AT E299189T1
Authority
AT
Austria
Prior art keywords
splicing
acceptor site
functional splice
retroviral
vector
Prior art date
Application number
AT98944085T
Other languages
English (en)
Inventor
Chris Bebbington
Susan Kingsman
Mark Uden
Alan Kingsman
Kyriacos Mitrophanos
Original Assignee
Oxford Biomedica Ltd
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 Oxford Biomedica Ltd filed Critical Oxford Biomedica Ltd
Application granted granted Critical
Publication of ATE299189T1 publication Critical patent/ATE299189T1/de

Links

Classifications

    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20111—Lyssavirus, e.g. rabies virus
    • C12N2760/20122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
    • 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6045—RNA rev transcr viruses
    • C12N2810/6054—Retroviridae
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Prostheses (AREA)
  • Materials For Medical Uses (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Cleaning Implements For Floors, Carpets, Furniture, Walls, And The Like (AREA)
  • Mattresses And Other Support Structures For Chairs And Beds (AREA)
  • Adhesives Or Adhesive Processes (AREA)
AT98944085T 1997-09-25 1998-09-23 Retrovirale vektoren, die funktionelle spleiss- donor- und spleiss-akzeptor-stellen enthalten ATE299189T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB9720465.5A GB9720465D0 (en) 1997-09-25 1997-09-25 Dual-virus vectors
PCT/GB1998/002867 WO1999015683A1 (en) 1997-09-25 1998-09-23 Retroviral vectors comprising a functional splice donor site and a functional splice acceptor site

Publications (1)

Publication Number Publication Date
ATE299189T1 true ATE299189T1 (de) 2005-07-15

Family

ID=10819668

Family Applications (1)

Application Number Title Priority Date Filing Date
AT98944085T ATE299189T1 (de) 1997-09-25 1998-09-23 Retrovirale vektoren, die funktionelle spleiss- donor- und spleiss-akzeptor-stellen enthalten

Country Status (14)

Country Link
US (2) US6808922B1 (de)
EP (1) EP1017837B1 (de)
JP (1) JP2001517452A (de)
KR (1) KR20010052077A (de)
CN (1) CN1203183C (de)
AT (1) ATE299189T1 (de)
AU (1) AU750110B2 (de)
CA (1) CA2304259A1 (de)
DE (1) DE69830798T2 (de)
GB (1) GB9720465D0 (de)
IL (1) IL134896A0 (de)
NO (1) NO20001486L (de)
NZ (1) NZ503317A (de)
WO (1) WO1999015683A1 (de)

Families Citing this family (167)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6348450B1 (en) 1997-08-13 2002-02-19 The Uab Research Foundation Noninvasive genetic immunization, expression products therefrom and uses thereof
GB2356200B (en) * 1997-12-22 2002-05-01 Oxford Biomedica Ltd Retroviral vectors
ES2373406T3 (es) * 1997-12-22 2012-02-03 Oxford Biomedica (Uk) Limited Vectores basados en el virus de la anemia infecciosa equina (vaie).
EP1036091B2 (de) 1998-11-18 2008-03-26 Oxford Biomedica (UK) Limited Verwendung vom krebs-assoziierten antigen 5t4 zur immuntherapie
US7148035B1 (en) 1998-11-18 2006-12-12 Oxford Biomedica (Uk) Limited Polypeptide
GB0400443D0 (en) 2004-01-09 2004-02-11 Oxford Biomedica Ltd Cascade
GB9906615D0 (en) * 1999-03-22 1999-05-19 Oxford Biomedica Ltd Vector
US6979568B1 (en) 1999-06-22 2005-12-27 Dnavec Research, Inc. Vector for the expression of two foreign genes
CN1321182C (zh) * 1999-12-30 2007-06-13 卫生部艾滋病预防与控制中心 马传染性贫血病毒代表毒株的全基因克隆及其应用
GB0009760D0 (en) 2000-04-19 2000-06-07 Oxford Biomedica Ltd Method
GB0024550D0 (de) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US6573092B1 (en) 2000-10-10 2003-06-03 Genvec, Inc. Method of preparing a eukaryotic viral vector
CA2344208A1 (en) 2001-04-30 2002-10-30 Oxford Biomedica (Uk) Limited Method
SI1504108T1 (sl) 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviralni vektor
GB0203419D0 (en) 2002-02-13 2002-04-03 Oxford Biomedica Ltd Peptide
GB0203420D0 (en) 2002-02-13 2002-04-03 Oxford Biomedica Ltd Peptide
CN101163494A (zh) * 2005-02-23 2008-04-16 Uab研究基金会 烷基糖苷增强的接种
CA2606287A1 (en) 2005-05-13 2006-11-16 Oxford Biomedica (Uk) Limited Peptide
GB0526210D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Vectors
US8377691B2 (en) * 2006-01-26 2013-02-19 Integral Molecular, Inc. Lipoparticles comprising ion channels, methods of making and using the same
WO2007089780A2 (en) * 2006-01-30 2007-08-09 Licentia, Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
DK2307551T3 (en) 2008-06-18 2017-03-20 Oxford Biomedica (Uk) Ltd CLEANING RETROVIRAL VECTORS
WO2011148194A1 (en) 2010-05-28 2011-12-01 Oxford Biomedica (Uk) Ltd Delivery of lentiviral vectors to the brain
GB201118636D0 (en) 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
EP3031921B1 (de) 2012-12-12 2025-03-12 The Broad Institute, Inc. Ausgabe, bearbeitung und optimierung von systeme, verfahren und zusammensetzungen für sequenzmanipulation und therapeutische anwendungen
EP2931899A1 (de) 2012-12-12 2015-10-21 The Broad Institute, Inc. Funktionale genomik unter verwendung von crispr-cas systemen, zusammensetzungen, verfahren, knockout-bibliotheken und anwendungen davon
AU2014255665B2 (en) * 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
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
WO2015053398A1 (ja) * 2013-10-11 2015-04-16 タカラバイオ株式会社 高力価レトロウイルスベクター
GB201318804D0 (en) 2013-10-24 2013-12-11 Adaptimmune Ltd Vectors for transgene expression
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
JP7103750B2 (ja) 2013-12-12 2022-07-20 ザ・ブロード・インスティテュート・インコーポレイテッド ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用
EP3080259B1 (de) 2013-12-12 2023-02-01 The Broad Institute, Inc. Konstruktion von systemen, verfahren und optimierte führungszusammensetzungen mit neuen architekturen zur sequenzmanipulation
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
GB201322574D0 (en) 2013-12-19 2014-02-05 Equigerminal Sa Retroviral peptides
KR20230076867A (ko) 2013-12-20 2023-05-31 더 브로드 인스티튜트, 인코퍼레이티드 신생항원 백신과의 병용 요법
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
EP3212788A2 (de) 2014-10-27 2017-09-06 The Broad Institute, Inc. Zusammensetzungen, verfahren und verwendung von screening auf synthetische tödliche gene
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3889260A1 (de) 2014-12-12 2021-10-06 The Broad Institute, Inc. Geschützte guide-rnas (pgrnas)
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
EP3234130B1 (de) 2014-12-19 2020-11-25 The Broad Institute, Inc. Verfahren zum profiling vom t-zell-rezeptor-repertoire
EP3234192B1 (de) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbeeinflusste identifizierung von doppelstrangbrüchen und genomischer umordnung durch genomweite einsatzerfassungssequenzierung
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
SI3265571T1 (sl) * 2015-03-03 2022-10-28 Fondazione Telethon Več vektorski sistem in njegove uporabe
EP3268044A2 (de) 2015-03-11 2018-01-17 The Broad Institute Inc. Prmt5-inhibitoren zur behandlung von krebs mit reduzierter mtap-aktivität
IL294183B2 (en) 2015-05-20 2023-10-01 Dana Farber Cancer Inst Inc shared neoantigens
EP3822291A1 (de) 2015-06-10 2021-05-19 The Broad Institute Inc. Antikörper, verbindungen und tests zur identifizierung und behandlung von kachexie oder präkachexie
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
TWI906646B (zh) 2015-06-18 2025-12-01 美商博得學院股份有限公司 降低脫靶效應的ｃｒｉｓｐｒ酶突變
WO2017031370A1 (en) 2015-08-18 2017-02-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
EP3368687B1 (de) 2015-10-27 2021-09-29 The Broad Institute, Inc. Zusammensetzungen und verfahren gegen krebsspezifische sequenzvariationen
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
AU2017254477A1 (en) 2016-04-18 2018-11-01 Jennifer G. ABELIN Improved HLA epitope prediction
AU2017257274B2 (en) 2016-04-19 2023-07-13 Massachusetts Institute Of Technology Novel CRISPR enzymes and systems
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
EP3445853A1 (de) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1-komplexe mit reduzierter indelaktivität
JP2019519250A (ja) 2016-05-10 2019-07-11 ユナイテッド ステイツ ガバメント アズ リプレゼンテッド バイ ザ デパートメント オブ ベテランズ アフェアーズＵｎｉｔｅｄ Ｓｔａｔｅｓ Ｇｏｖｅｒｎｍｅｎｔ Ａｓ Ｒｅｐｒｅｓｅｎｔｅｄ Ｂｙ Ｔｈｅ Ｄｅｐａｒｔｍｅｎｔ Ｏｆ Ｖｅｔｅｒａｎｓ Ａｆｆａｉｒｓ Ｈｉｖ−１感染と複製に必須な遺伝子を切断するｃｒｉｓｐｒ／ｃａｓの構築物のレンチウィルスによる送達
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
EP4485466A3 (de) 2016-08-17 2025-04-02 The Broad Institute Inc. Neuartige crispr-enzyme und systeme
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
US11549149B2 (en) 2017-01-24 2023-01-10 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
CA3052099A1 (en) 2017-01-30 2018-08-02 Mathias LABS Repair template linkage to endonucleases for genome engineering
US11965892B2 (en) 2017-02-12 2024-04-23 Biontech Us Inc. HLA-based methods and compositions and uses thereof
JP2020511141A (ja) 2017-03-15 2020-04-16 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１３ｂオルソログＣＲＩＳＰＲ酵素及び系
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
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
WO2018213726A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US12415000B2 (en) 2017-07-07 2025-09-16 The Broad Institute, Inc. CRISPR system based antiviral therapy
CN107502594A (zh) * 2017-07-14 2017-12-22 中国药科大学 一种稳定表达外源性ex－4基因的骨髓源间充质干细胞株
AU2018338318B2 (en) 2017-09-21 2022-12-22 Massachusetts Institute Of Technology Systems, methods, and compositions for targeted nucleic acid editing
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
EP3710039A4 (de) 2017-11-13 2021-08-04 The Broad Institute, Inc. Verfahren und zusammensetzungen zur behandlung von krebs durch targeting des clec2d-klrb1-wegs
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
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
SG11202011015QA (en) 2018-05-15 2020-12-30 Flagship Pioneering Innovations V Inc Fusosome compositions and uses thereof
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
JP2021532815A (ja) 2018-08-07 2021-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 新規Ｃａｓ１２ｂ酵素およびシステム
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
WO2020072700A1 (en) 2018-10-02 2020-04-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
AU2019378883A1 (en) 2018-11-14 2021-06-03 Flagship Pioneering Innovations V, Inc. Fusosome compositions for T cell delivery
US20220008557A1 (en) 2018-11-14 2022-01-13 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
EP3880832A1 (de) 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosomzusammensetzungen zur verabreichung von hämatopoetischen stammzellen
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
CN113544266A (zh) 2018-12-17 2021-10-22 博德研究所 Crispr相关转座酶系统和其使用方法
KR20240091046A (ko) 2018-12-21 2024-06-21 바이오엔테크 유에스 인크. Hla 클래스 ii-특이적 에피토프 예측 및 cd4+ t 세포 특징화를 위한 방법 및 시스템
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (de) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von sensorineuralem hörverlust unter verwendung von otoferlin-doppelvektorsystemen
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US20220175962A1 (en) 2019-03-10 2022-06-09 Sio Gene Therapies Inc. Gene therapy compositions and methods for treating parkinson's disease
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
US12390018B2 (en) 2019-05-20 2025-08-19 Shanna Dickinson Convertible crib
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
CN115023499A (zh) 2019-08-16 2022-09-06 麻省理工学院 使用CRISPR/Cas13的靶向反式剪接
CA3152525A1 (en) 2019-09-03 2021-03-11 Sana Biotechnology, Inc. Cd24-associated particles and related methods and uses thereof
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US12394502B2 (en) 2019-10-02 2025-08-19 The General Hospital Corporation Method for predicting HLA-binding peptides using protein structural features
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
CA3178308A1 (en) 2020-03-31 2021-10-07 Sana Biotechnology, Inc. Targeted lipid particles and compositions and uses thereof
US20230190871A1 (en) 2020-05-20 2023-06-22 Sana Biotechnology, Inc. Methods and compositions for treatment of viral infections
WO2022047316A1 (en) 2020-08-28 2022-03-03 Sana Biotechnology, Inc. Modified anti-viral binding agents
JP2024501482A (ja) 2020-12-14 2024-01-12 ビオンテック ユーエス インコーポレイテッド がん免疫療法のための組織特異的抗原
JP2024501971A (ja) 2020-12-31 2024-01-17 サナ バイオテクノロジー，インコーポレイテッド Ｃａｒ－ｔ活性を調節するための方法及び組成物
MX2023008081A (es) 2021-01-11 2023-09-12 Sana Biotechnology Inc Uso de vectores virales dirigidos a cd8.
CN117529491A (zh) * 2021-04-23 2024-02-06 阿西莫夫公司 用于慢病毒载体产生的稳定产生系统
JP2024521811A (ja) 2021-05-28 2024-06-04 サナ バイオテクノロジー，インコーポレイテッド 短縮型ヒヒ内在性レトロウイルス（ＢａＥＶ）エンベロープ糖タンパク質を含む脂質粒子、ならびに関連する方法及び使用
KR20240046319A (ko) 2021-07-14 2024-04-08 사나 바이오테크놀로지, 인크. 저면역원성 세포에서의 y 염색체-연결 항원의 변경된 발현
EP4381081A1 (de) 2021-08-04 2024-06-12 Sana Biotechnology, Inc. Verwendung von auf cd4 gerichteten viralen vektoren
US20250230511A1 (en) 2021-10-29 2025-07-17 Sana Biotechnology, Inc. Methods and reagents for amplifying viral vector nucleic acid products
GB202117405D0 (en) 2021-12-02 2022-01-19 Univ London Queen Mary Peptide
WO2023114949A1 (en) 2021-12-16 2023-06-22 Sana Biotechnology, Inc. Methods and systems of particle production
US20250059239A1 (en) 2021-12-17 2025-02-20 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
EP4463135A2 (de) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Verfahren zur ex-vivo-dosierung und -verabreichung von lipidpartikeln oder viralen vektoren sowie entsprechende systeme und verwendungen
EP4472646A1 (de) 2022-02-01 2024-12-11 Sana Biotechnology, Inc. Auf cd3 abzielende lentivirale vektoren und verwendungen davon
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
EP4479416A1 (de) 2022-02-17 2024-12-25 Sana Biotechnology, Inc. Manipulierte cd47-proteine und verwendungen davon
WO2023183313A1 (en) 2022-03-22 2023-09-28 Sana Biotechnology, Inc. Engineering cells with a transgene in b2m or ciita locus and associated compositions and methods
WO2023193015A1 (en) 2022-04-01 2023-10-05 Sana Biotechnology, Inc. Cytokine receptor agonist and viral vector combination therapies
IL316038A (en) 2022-04-04 2024-11-01 Univ California Preparations and methods for genetic complementation
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
US20260055146A1 (en) 2022-08-24 2026-02-26 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024064838A1 (en) 2022-09-21 2024-03-28 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
EP4698666A1 (de) 2023-04-18 2026-02-25 Sana Biotechnology, Inc. Universelle protein-g-fusogene und adaptersysteme davon sowie zugehörige lipidpartikel und verwendungen
WO2024220597A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Digital droplet based assay for detecting replication competent lentiviral vector
EP4716750A1 (de) 2023-05-23 2026-04-01 Sana Biotechnology, Inc. Tandem-fusogene und verwandte lipidpartikel
US12531162B1 (en) * 2023-05-31 2026-01-20 Northeastern University Multi-dimensional phenotypic space for genotype to phenotype mapping and intelligent design of cancer drug therapies using a deep learning net
WO2025043172A1 (en) 2023-08-23 2025-02-27 Sana Biotechnology, Inc. Modified cd47 proteins and their uses
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
US12562256B2 (en) * 2023-11-07 2026-02-24 New York University Systems, methods and computer-accessible medium for identifying target pairs for CAR-T therapy
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods
WO2025212519A1 (en) 2024-04-01 2025-10-09 Moonlight Bio, Inc. Dll3 binding proteins and uses thereof
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6544771B1 (en) 1987-12-11 2003-04-08 Cell Genesys, Inc. Retroviral gene therapy vectors and therapeutic methods based thereon
US5631162A (en) 1993-06-11 1997-05-20 Massachusetts Institute Of Technology Retroviral vectors for transducing β-globin gene and β-locus control region derivatives
WO1996028563A1 (en) 1995-03-09 1996-09-19 Bavarian Nordic Vectors carrying therapeutic genes encoding antimicrobial peptides for gene therapy
EP0932693A1 (de) * 1996-10-10 1999-08-04 Henrik Garoff Alphavirus-retrovirus vektoren

Also Published As

Publication number Publication date
IL134896A0 (en) 2001-05-20
AU9175698A (en) 1999-04-12
CN1203183C (zh) 2005-05-25
US7303910B2 (en) 2007-12-04
WO1999015683A8 (en) 1999-05-06
WO1999015683A1 (en) 1999-04-01
DE69830798T2 (de) 2005-12-22
GB9720465D0 (en) 1997-11-26
NO20001486L (no) 2000-05-25
DE69830798D1 (de) 2005-08-11
US6808922B1 (en) 2004-10-26
AU750110B2 (en) 2002-07-11
NO20001486D0 (no) 2000-03-22
CN1279723A (zh) 2001-01-10
EP1017837B1 (de) 2005-07-06
US20050009186A1 (en) 2005-01-13
NZ503317A (en) 2002-05-31
KR20010052077A (ko) 2001-06-25
EP1017837A1 (de) 2000-07-12
CA2304259A1 (en) 1999-04-01
JP2001517452A (ja) 2001-10-09

Similar Documents

Publication Publication Date Title
ATE299189T1 (de) Retrovirale vektoren, die funktionelle spleiss- donor- und spleiss-akzeptor-stellen enthalten
PT950386E (pt) Stent com administracao local de rapamicina
DE69738218D1 (de) Cvd von dünnen fluorkohlenstoffen-polymer-filmen
KR920000967A (ko) 질화 규소막의 형성방법
TR199701146T1 (xx) N�rotensin resept�rleri �zerinde etkili 1-Fenilpirazol-3-Karboksamidler.
ES2189851T3 (es) Copolimero tribloque polisiloxano-polioxietileno-polioxipropileno y composiciones antiespumantes que contienen los mismos.
GEP19991571B (en) Substituted monocyclicpentadienyl - metall complex, a method of its reception and a method of reception of (co) polymers of ethyl
TR199801127T2 (xx) Yeni 5-Amino-3-siyano-etils�lfinil-1-fenil-pirazol bile�ikleri, bunlar�n pestisit olarak kullan�lmas�.
ES2176414T3 (es) Terminal de radiocomunicacion preparado para recibir una cubierta de proteccion.
TR199800773T2 (xx) Trombin inhibit�rleri.
DE69206093D1 (de) Kosmetisches Mittel.
EP0730550A4 (de) Verriegelung für kartonzuschnitt
DE69529729D1 (de) Synthetisierer hoher Präzision sowie niedrigen Phasenrauschens, mit Vektormodulator
DK0984778T3 (da) Neuropeptid-Y-ligander
MY121808A (en) Module for interconnecting two monopair lines
ATE308548T1 (de) Glycerophosphoinositol-derivate als cytosolic a2 phospholipase modulatoren
KR910007469A (ko) 비디오 카메라 및 부속품 운반용 케이스
GB2362886A (en) Retroviral vectors comprising functional and non-functional splice donor and splice acceptor sites
GB2344592A (en) Retroviral vectors comprising a functional splice donor site and a funcional splice acceptor site
DE69909713D1 (de) Integriert-optisches Bauelement
DK0723948T3 (da) Capsaicinoidholdig tårefremkalder
DE69609390D1 (de) Einspritzapparat für Kohlenwasserstoff-Einsätze
HUP9901101A2 (hu) Stabil peroxigénes fehérítőt tartalmazó készítmények
FR2700724B1 (fr) Etui pour une série d'outils, notamment de clés mâles coudées.
BR9909046A (pt) Circuito demodulador

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1017837

Country of ref document: EP

REN Ceased due to non-payment of the annual fee