AU2001292093A1 - Retroviral vectors containing internal ribosomal entry sites - Google Patents

Retroviral vectors containing internal ribosomal entry sites

Info

Publication number
AU2001292093A1
AU2001292093A1 AU2001292093A AU9209301A AU2001292093A1 AU 2001292093 A1 AU2001292093 A1 AU 2001292093A1 AU 2001292093 A AU2001292093 A AU 2001292093A AU 9209301 A AU9209301 A AU 9209301A AU 2001292093 A1 AU2001292093 A1 AU 2001292093A1
Authority
AU
Australia
Prior art keywords
vectors containing
retroviral vectors
entry sites
containing internal
ribosomal entry
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2001292093A
Inventor
Mimoun Azzouz
Alan John Kingsman
Enca Martin-Rendon
Nicholas D. Mazarakis
Jonathan Rohll
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Oxford Biomedica UK Ltd
Original Assignee
Oxford Biomedica UK 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 UK Ltd filed Critical Oxford Biomedica UK Ltd
Publication of AU2001292093A1 publication Critical patent/AU2001292093A1/en
Abandoned legal-status Critical Current

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • 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
    • 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
    • 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
    • C12N2840/206—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES having multiple IRES

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (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)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Molds, Cores, And Manufacturing Methods Thereof (AREA)

Abstract

The present invention relates to retroviral vector genomes and to vector systems comprising such genomes. In particular the present invention relates to a retroviral vector genome comprising two or more NOIs operably linked by one or more Internal Ribosome Entry Site(s); a lentiviral vector genome comprising two or more NOIs suitable for treating a neurodegenerative disorder; and a lentiviral vector genome which encodes tyrosine hydroxylase, GTP-cyclohydrolase I and optionally Aromatic Amino Acid Dopa Decarboxylase.
AU2001292093A 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites Abandoned AU2001292093A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB0024550.6A GB0024550D0 (en) 2000-10-06 2000-10-06
GB0024550.6 2000-10-06
PCT/GB2001/004433 WO2002029065A2 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites

Publications (1)

Publication Number Publication Date
AU2001292093A1 true AU2001292093A1 (en) 2002-04-15

Family

ID=9900826

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001292093A Abandoned AU2001292093A1 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites

Country Status (13)

Country Link
US (3) US7259015B2 (en)
EP (2) EP2180057A1 (en)
JP (1) JP4224295B2 (en)
AT (1) ATE474058T1 (en)
AU (1) AU2001292093A1 (en)
CA (1) CA2424738A1 (en)
CY (1) CY1110817T1 (en)
DE (1) DE60142576D1 (en)
DK (1) DK1337655T3 (en)
ES (1) ES2348277T3 (en)
GB (1) GB0024550D0 (en)
PT (1) PT1337655E (en)
WO (1) WO2002029065A2 (en)

Families Citing this family (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7588757B2 (en) * 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
PT1504108E (en) * 2002-02-01 2013-06-12 Oxford Biomedica Ltd Lentiviral vector
AU2007353396C1 (en) * 2006-11-02 2013-06-27 Biomolecular Holdings Llc Hybrid immunoglobulins with moving parts
PL2307551T3 (en) 2008-06-18 2017-07-31 Oxford Biomedica (Uk) Limited Purification of retroviral vectors
US8829173B2 (en) * 2008-09-26 2014-09-09 Tocagen Inc. Recombinant vectors
US20110269826A1 (en) * 2008-11-11 2011-11-03 Oxford Biomedica (Uk) Limited Method
BR112012010866A2 (en) * 2009-11-09 2016-11-29 Genepod Therapeutics Ab "New viral vector construct for continuous optimized specific synthesis of dopa by neurons in vivo."
CN102946907A (en) * 2010-05-28 2013-02-27 牛津生物医学（英国）有限公司 Delivery of lentiviral vectors to the brain
WO2012135389A2 (en) * 2011-03-28 2012-10-04 The Regents Of The University Of California Host cells and methods for oxidizing aromatic amino acids
AU2012216792A1 (en) 2011-09-12 2013-03-28 International Aids Vaccine Initiative Immunoselection of recombinant vesicular stomatitis virus expressing HIV-1 proteins by broadly neutralizing antibodies
EP2586461A1 (en) 2011-10-27 2013-05-01 Christopher L. Parks Viral particles derived from an enveloped virus
US10400252B2 (en) 2011-10-28 2019-09-03 Oxford Biomedica (Uk) Ltd. Catecholamine enzyme fusions
JP6698114B2 (en) * 2011-10-28 2020-05-27 オックスフォード バイオメディカ （ユーケー） リミテッド Construction
GB201118636D0 (en) * 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
US9347065B2 (en) 2012-03-29 2016-05-24 International Aids Vaccine Initiative Methods to improve vector expression and genetic stability
EP2848687B1 (en) 2012-04-27 2017-09-13 JCR Pharmaceuticals Co., Ltd. Novel expression vector
EP2895611A4 (en) * 2012-09-14 2016-04-27 Univ California Lentiviral vector for stem cell gene therapy of sickle cell disease
EP2909324B1 (en) 2012-10-25 2020-02-26 Tocagen Inc. Retroviral vector with mini-promoter cassette
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
US10106816B2 (en) 2012-12-14 2018-10-23 Case Western Reserve University Genomic RNA packaging enhancer element
BR112015031608A2 (en) 2013-06-17 2017-08-22 Massachusetts Inst Technology APPLICATION AND USE OF CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR LIVER TARGETING AND THERAPY
EP3011032B1 (en) 2013-06-17 2019-10-16 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
RU2716421C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery, use and use in therapy of crispr-cas systems and compositions for targeted action on disorders and diseases using viral components
WO2015048690A1 (en) * 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
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
US11725237B2 (en) 2013-12-05 2023-08-15 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
SG10201804974RA (en) 2013-12-12 2018-07-30 Broad Inst Inc Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
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
BR112016013201B1 (en) 2013-12-12 2023-01-31 The Broad Institute, Inc. USE OF A COMPOSITION COMPRISING A CRISPR-CAS SYSTEM IN THE TREATMENT OF A GENETIC OCULAR DISEASE
WO2015095811A2 (en) 2013-12-20 2015-06-25 The Board Institute Inc. Combination therapy with neoantigen vaccine
EP3686279B1 (en) 2014-08-17 2023-01-04 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
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
WO2016073693A2 (en) * 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3230452A1 (en) 2014-12-12 2017-10-18 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
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)
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
EP3757211A1 (en) 2014-12-19 2020-12-30 The Broad Institute, Inc. Methods for profiling the t-cell-receptor repertoire
WO2016100975A1 (en) 2014-12-19 2016-06-23 Massachsetts Institute Ot Technology Molecular biomarkers for cancer immunotherapy
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
ES2578903B1 (en) * 2015-01-02 2017-07-07 Instituto De Salud Carlos Iii LENTIVIRAL VECTOR OF SELF-LIMITED EXPRESSION
US20180271891A1 (en) 2015-03-11 2018-09-27 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
CA2986235A1 (en) 2015-05-20 2016-11-24 The Broad Institute, Inc. Shared neoantigens
EP3822291A1 (en) 2015-06-10 2021-05-19 The Broad Institute Inc. Antibodies, compounds and screens for identifying and treating cachexia or pre-cachexia
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
CA2989830A1 (en) 2015-06-18 2016-12-22 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
EP3436575A1 (en) 2015-06-18 2019-02-06 The Broad Institute Inc. Novel crispr enzymes and systems
RU2018104098A (en) * 2015-08-03 2019-09-06 Майодопа Лимитед SYSTEMIC SYNTHESIS AND REGULATION OF L-DOPA
EP3337908A4 (en) 2015-08-18 2019-01-23 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
US20190255107A1 (en) 2015-10-09 2019-08-22 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP3365441A1 (en) 2015-10-22 2018-08-29 The Broad Institute Inc. Type vi-b crispr enzymes and systems
EP3368687B1 (en) 2015-10-27 2021-09-29 The Broad Institute, Inc. Compositions and methods for targeting cancer-specific sequence variations
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
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
US20190233814A1 (en) 2015-12-18 2019-08-01 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190346442A1 (en) 2016-04-18 2019-11-14 The Broad Institute, Inc. Improved hla epitope prediction
CN110382692A (en) 2016-04-19 2019-10-25 博德研究所 Novel C RISPR enzyme and system
WO2017189308A1 (en) 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems
WO2017184786A1 (en) 2016-04-19 2017-10-26 The Broad Institute Inc. Cpf1 complexes with reduced indel activity
US11286501B2 (en) * 2016-04-20 2022-03-29 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas O.A, M.P. Methods of treating or preventing pyruvate kinase deficiency
JP7267013B2 (en) 2016-06-17 2023-05-01 ザ・ブロード・インスティテュート・インコーポレイテッド Type VI CRISPR orthologs and systems
US20210222164A1 (en) 2016-06-29 2021-07-22 The Broad Institute, Inc. Crispr-cas systems having destabilization domain
CA3032418C (en) * 2016-08-01 2024-02-20 Health Research, Inc. Compositions and methods for rapid cloning of t-cell receptors
CN110312799A (en) 2016-08-17 2019-10-08 博德研究所 Novel C RISPR enzyme and system
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. 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
WO2018049273A1 (en) * 2016-09-08 2018-03-15 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia
US20200016202A1 (en) 2016-10-07 2020-01-16 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
MX2019004487A (en) 2016-10-20 2020-02-07 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease.
WO2018140391A1 (en) 2017-01-24 2018-08-02 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
BR112019015578A2 (en) 2017-01-30 2020-03-10 KWS SAAT SE & Co. KGaA CONNECTION OF ENDONUCLEASE REPAIR MODEL FOR GENOME ENGINEERING
CN116693695A (en) 2017-02-12 2023-09-05 百欧恩泰美国公司 HLA-based methods and compositions and uses thereof
WO2018170333A1 (en) 2017-03-15 2018-09-20 The Broad Institute, Inc. Novel cas13b orthologues crispr enzymes and systems
EP3610009A1 (en) 2017-04-12 2020-02-19 The Broad Institute, Inc. Novel type vi crispr orthologs and systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
WO2018204777A2 (en) 2017-05-05 2018-11-08 The Broad Institute, Inc. Methods for identification and modification of lncrna associated with target genotypes and phenotypes
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN111511388A (en) 2017-09-21 2020-08-07 博德研究所 Systems, methods, and compositions for targeted nucleic acid editing
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
CA3076253A1 (en) 2017-10-16 2019-04-25 Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicaso.A., M.P. Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US20210363260A1 (en) 2017-11-13 2021-11-25 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
JP7197901B2 (en) * 2018-01-11 2022-12-28 学校法人慈恵大学 Vector plasmids, lentiviral vector systems, cells and cell preparations used in gene therapy for Hunter syndrome
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
CN116024271A (en) * 2018-05-31 2023-04-28 康霖生物科技（杭州）有限公司 Gene sequence construct for treating central nervous system diseases
CA3106035A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Cas12b enzymes and systems
WO2020041380A1 (en) 2018-08-20 2020-02-27 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
EP3898958A1 (en) 2018-12-17 2021-10-27 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
WO2020132586A1 (en) 2018-12-21 2020-06-25 Neon Therapeutics, Inc. Method and systems for prediction of hla class ii-specific epitopes and characterization of cd4+ t cells
SG11202107325TA (en) * 2019-01-04 2021-08-30 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease
WO2020183374A1 (en) 2019-03-10 2020-09-17 Axovant Sciences Gmbh Gene therapy compositions and methods for treating parkinson's disease
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
AU2020333667A1 (en) 2019-08-16 2022-02-24 Massachusetts Institute Of Technology Targeted trans-splicing using CRISPR/Cas13
KR20230043869A (en) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Placophilin-2 (PKP2) gene therapy using AAV vectors
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
US20230374542A1 (en) 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)
EP4259206A2 (en) 2020-12-14 2023-10-18 BioNTech US Inc. Tissue-specific antigens for cancer immunotherapy
CN114107396B (en) * 2021-11-26 2024-02-02 和元智造(上海)基因技术有限公司 Lentiviral vector, system and application thereof
WO2023196818A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California Genetic complementation compositions and methods
WO2024010067A1 (en) * 2022-07-08 2024-01-11 Ｊｃｒファーマ株式会社 Nuecleic acid molecule, vector, recombinant cells, and drug for treating central nervous system diseases
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5169762A (en) 1983-03-03 1992-12-08 Genentech, Inc. Human nerve growth factor by recombinant technology
WO1991000047A1 (en) 1989-06-30 1991-01-10 The Regents Of The University Of California Retrovirus detection
JPH06500923A (en) 1990-09-21 1994-01-27 カイロン　コーポレイション packaging cells
DE69121208T2 (en) 1990-09-25 1997-01-23 Genentech Inc NEW NEUROTROPICAL FACTOR
DE69232438T2 (en) 1991-08-07 2002-10-10 W French Anderson INTERNAL RIBOSOM INPUTS INCLUDING RETROVIRAL VECTORS
WO1994024870A1 (en) * 1993-01-20 1994-11-10 Biotransplant, Inc. Retroviral vectors capable of expressing multimeric proteins from multiple translational initiation sites
US5834256A (en) 1993-06-11 1998-11-10 Cell Genesys, Inc. Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells
US5728803A (en) 1994-06-03 1998-03-17 Genentech, Inc. Pantropic neurotrophic factors
US6103226A (en) * 1994-08-12 2000-08-15 Arch Development Corporation Genetically engineered cells that produce produce L. Dopa
WO1996009400A1 (en) 1994-09-19 1996-03-28 Systemix, Inc. Methods for genetically modifying hematopoietic stem cells
US5739307A (en) 1995-08-28 1998-04-14 Washington University Polynucleotide encoding neurturin neurotrophic factor
EP0857217B1 (en) 1995-10-16 2005-04-13 Dana-Farber Cancer Institute Novel expression vectors and methods of use
CA2237000A1 (en) 1995-11-08 1997-05-15 The Whitehead Institute For Biomedical Research Stable packaging cell line producing pseudotyped retroviruses
WO1997018319A1 (en) 1995-11-14 1997-05-22 Somatix Therapy Corporation Joint expression of gtp cyclohydrolase and tyrosine hydroxylase
US6222022B1 (en) 1996-03-14 2001-04-24 Washington University Persephin and related growth factors
US5739018A (en) 1996-08-07 1998-04-14 The Regents Of The University Of California Packaging cell lines for pseudotyped retroviral vectors
US6133027A (en) 1996-08-07 2000-10-17 City Of Hope Inducible expression system
WO1998012338A1 (en) 1996-09-21 1998-03-26 Viromedica Pacific Limited Improved retroviral vectors for gene therapy
HUP0000421A2 (en) 1996-10-17 2000-06-28 Oxford Biomedica (Uk) Limited Retroviral vectors
US5969105A (en) 1996-10-25 1999-10-19 Feng; Yiqing Stem cell factor receptor agonists
GB9622500D0 (en) 1996-10-29 1997-01-08 Oxford Biomedica Ltd Therapeutic gene
NZ500740A (en) * 1997-05-13 2001-02-23 Univ North Carolina Recombinant lentivirus-based gene transfer vectors comprising 3 vectors from Equine Infectious Anemia Virus (EIAV)
US5834914A (en) 1997-09-08 1998-11-10 Safetran Systems Corporation Railroad crossing gate mechanism control system
US6136597A (en) * 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
AU747609B2 (en) * 1997-09-23 2002-05-16 Oxford Biomedica (Uk) Limited Expression of genes in hematopoietic stem cells in hischaemic conditions
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
EP1042493A1 (en) 1997-12-22 2000-10-11 Oxford Biomedica (UK) Limited Equine infectious anaemia virus (eiav) based retroviral vectors
GB2356200B (en) * 1997-12-22 2002-05-01 Oxford Biomedica Ltd Retroviral vectors
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
DE69941049D1 (en) 1998-05-22 2009-08-13 Oxford Biomedica Ltd RETROVIRAL ADMINISTRATION SYSTEM
US6555342B1 (en) * 1998-06-03 2003-04-29 Uab Research Foundation Fusion protein delivery system and uses thereof
US6958226B1 (en) * 1998-09-11 2005-10-25 The Children's Medical Center Corp. Packaging cells comprising codon-optimized gagpol sequences and lacking lentiviral accessory proteins
US20020002269A1 (en) 1998-09-29 2002-01-03 Jeffrey D. Milbrandt Artemin, a neurotrophic factor
US6506378B1 (en) * 1998-12-16 2003-01-14 Arch Development Corporation Vesicular monoamine transporter gene therapy in Parkinson's disease

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