WO2002029065A3 - Retroviral vectors containing internal ribosomal entry sites - Google Patents

Retroviral vectors containing internal ribosomal entry sites Download PDF

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Publication number
WO2002029065A3
WO2002029065A3 PCT/GB2001/004433 GB0104433W WO0229065A3 WO 2002029065 A3 WO2002029065 A3 WO 2002029065A3 GB 0104433 W GB0104433 W GB 0104433W WO 0229065 A3 WO0229065 A3 WO 0229065A3
Authority
WO
WIPO (PCT)
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.)
Ceased
Application number
PCT/GB2001/004433
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French (fr)
Other versions
WO2002029065A2 (en
Inventor
Alan John Kingsman
Nicholas D Mazarakis
Enca Martin-Rendon
Mimoun Azzouz
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
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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
Priority to CA002424738A priority Critical patent/CA2424738A1/en
Priority to JP2002532634A priority patent/JP4224295B2/en
Priority to DE60142576T priority patent/DE60142576D1/en
Priority to AU2001292093A priority patent/AU2001292093A1/en
Priority to DK01972317.0T priority patent/DK1337655T3/en
Priority to AT01972317T priority patent/ATE474058T1/en
Priority to EP01972317A priority patent/EP1337655B1/en
Publication of WO2002029065A2 publication Critical patent/WO2002029065A2/en
Publication of WO2002029065A3 publication Critical patent/WO2002029065A3/en
Anticipated expiration legal-status Critical
Priority to US10/408,456 priority patent/US7259015B2/en
Priority to US10/873,573 priority patent/US7419829B2/en
Priority to US11/486,914 priority patent/US20070025970A1/en
Priority to US12/168,563 priority patent/US20090111106A1/en
Ceased 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)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (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.
PCT/GB2001/004433 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites Ceased WO2002029065A2 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
CA002424738A CA2424738A1 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites
JP2002532634A JP4224295B2 (en) 2000-10-06 2001-10-05 Vector system
DE60142576T DE60142576D1 (en) 2000-10-06 2001-10-05 LENTIVIRAL VECTORS FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
AU2001292093A AU2001292093A1 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites
DK01972317.0T DK1337655T3 (en) 2000-10-06 2001-10-05 Lentivirus vectors for the treatment of neurodegenerative diseases
AT01972317T ATE474058T1 (en) 2000-10-06 2001-10-05 LENTIVIRAL VECTORS FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES
EP01972317A EP1337655B1 (en) 2000-10-06 2001-10-05 Lentiviral vectors for the treatment of neurodegenerative diseases
US10/408,456 US7259015B2 (en) 2000-10-06 2003-04-07 Vector system
US10/873,573 US7419829B2 (en) 2000-10-06 2004-06-21 Vector system
US11/486,914 US20070025970A1 (en) 2000-10-06 2006-07-14 Vector system
US12/168,563 US20090111106A1 (en) 2000-10-06 2008-07-07 Vector System

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB0024550.6A GB0024550D0 (en) 2000-10-06 2000-10-06
GB0024550.6 2000-10-06

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US10/408,456 Continuation-In-Part US7259015B2 (en) 2000-10-06 2003-04-07 Vector system

Publications (2)

Publication Number Publication Date
WO2002029065A2 WO2002029065A2 (en) 2002-04-11
WO2002029065A3 true WO2002029065A3 (en) 2002-12-27

Family

ID=9900826

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2001/004433 Ceased WO2002029065A2 (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 (137)

* 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
SI1504108T1 (en) * 2002-02-01 2013-07-31 Oxford Biomedica (Uk) Limited Lentiviral vector
KR20090105913A (en) 2006-11-02 2009-10-07 다니엘 제이 카폰 Hybrid immunoglobulins with moving parts
DK2307551T3 (en) 2008-06-18 2017-03-20 Oxford Biomedica (Uk) Ltd CLEANING RETROVIRAL VECTORS
US8829173B2 (en) * 2008-09-26 2014-09-09 Tocagen Inc. Recombinant vectors
JP5559185B2 (en) * 2008-11-11 2014-07-23 オックスフォード バイオメディカ（ユーケー）リミテッド Method
KR20120089743A (en) * 2009-11-09 2012-08-13 제네포드 테라퓨틱스 에이비 Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo
WO2011148194A1 (en) 2010-05-28 2011-12-01 Oxford Biomedica (Uk) Ltd 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
CA2789539A1 (en) 2011-09-12 2013-03-12 International Aids Vaccine Initiative Immunoselection of recombinant vesicular stomatitis virus expressing hiv-1 proteins by broadly neutralizing antibodies
US9402894B2 (en) 2011-10-27 2016-08-02 International Aids Vaccine Initiative 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
US9657310B2 (en) 2012-04-27 2017-05-23 Jcr Pharmaceuticals Co., Ltd. Expression vector
JP2015529466A (en) * 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Lentiviral vector for stem cell gene therapy of sickle cell disease
ES2786051T3 (en) 2012-10-25 2020-10-08 Tocagen Inc Retroviral vector with mini-promoter cassette
EP3031921B1 (en) 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
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
WO2014093965A1 (en) * 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
RU2716420C2 (en) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Delivery and use of systems of crispr-cas, vectors and compositions for targeted action and therapy in liver
MX374532B (en) 2013-06-17 2025-03-06 Broad Inst Inc SUPPLY, USE AND THERAPEUTIC APPLICATIONS OF CRISPR-CAS SYSTEMS AND COMPOSITIONS, TO ACT ON DISORDERS AND DISEASES USING VIRAL COMPONENTS.
CN105683379A (en) 2013-06-17 2016-06-15 布罗德研究所有限公司 Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
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
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
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
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
JP7103750B2 (en) 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 Delivery, use and therapeutic application of CRISPR-Cas systems and compositions for genome editing
JP6793547B2 (en) 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 Optimization Function Systems, methods and compositions for sequence manipulation with the CRISPR-Cas system
JP6712948B2 (en) 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 Compositions and methods of using the CRISPR-cas system in nucleotide repeat disorders
KR20230076867A (en) 2013-12-20 2023-05-31 더 브로드 인스티튜트, 인코퍼레이티드 Combination therapy with neoantigen vaccine
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 (en) 2014-10-27 2017-09-06 The Broad Institute, Inc. Compositions, methods and use of synthetic lethal screening
RU2716991C2 (en) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Aadc polynucleotides for treating parkinson's disease
KR20230145206A (en) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
EP3889260A1 (en) 2014-12-12 2021-10-06 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)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
EP3234130B1 (en) 2014-12-19 2020-11-25 The Broad Institute, Inc. Methods for profiling the t-cell- receptor repertoire
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
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
ES2578903B1 (en) * 2015-01-02 2017-07-07 Instituto De Salud Carlos Iii LENTIVIRAL VECTOR OF SELF-LIMITED EXPRESSION
EP3268044A2 (en) 2015-03-11 2018-01-17 The Broad Institute Inc. Prmt5 inhibitors for the treatment of cancer with reduced mtap activty
IL294183B2 (en) 2015-05-20 2023-10-01 Dana Farber Cancer Inst 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
AU2016279077A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
TWI906646B (en) 2015-06-18 2025-12-01 美商博得學院股份有限公司 Crispr enzyme mutations reducing off-target effects
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US20190032079A1 (en) * 2015-08-03 2019-01-31 Myodopa Limited Systemic synthesis and regulation of l-dopa
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 (en) 2015-10-22 2023-09-29 布罗德研究所有限公司 VI-B type CRISPR enzyme and system
EP3368687B1 (en) 2015-10-27 2021-09-29 The Broad Institute, Inc. Compositions and methods for targeting cancer-specific sequence variations
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
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit 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
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR20260004568A (en) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 The novel CRISPR enzyme and system
HUE055862T2 (en) * 2016-04-20 2021-12-28 Centro De Investig Energeticas Compositions and methods for enhanced gene expression of PKLR
KR20240056729A (en) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
CN109642231A (en) 2016-06-17 2019-04-16 博德研究所 Type VI CRISPR orthologs and systems
US12595478B2 (en) 2016-06-29 2026-04-07 The Broad Institute, Inc. Crispr-Cas systems having destabilization domain
ES2877820T3 (en) * 2016-08-01 2021-11-17 Health Research Inc Compositions and methods for rapid cloning of T cell receptors
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
EP4485466A3 (en) 2016-08-17 2025-04-02 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
KR20240151726A (en) * 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Gene therapy for patients with fanconi anemia
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
KR102712926B1 (en) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 Methods and compositions for the treatment of Fabry disease
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 (en) 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 Novel Cas13b ortholog CRISPR enzyme and system
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
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US12415000B2 (en) 2017-07-07 2025-09-16 The Broad Institute, Inc. CRISPR system based antiviral therapy
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 altering function and structure of chromatin loops and/or domains
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
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
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
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR CANCER TREATMENT BY TARGETING THE CLEC2D-KLRB1 PATH
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
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
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (en) 2018-04-27 2021-01-29 Spacecraft Seven Llc GENE THERAPY FOR CNS DEGENERATION.
KR20210019996A (en) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. Composition and method for the treatment of Parkinson's disease
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
JP2021532815A (en) 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 New Cas12b enzyme and system
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
CN113544266A (en) 2018-12-17 2021-10-22 博德研究所 CRISPR-associated transposase system and method of use
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
KR20240091046A (en) 2018-12-21 2024-06-21 바이오엔테크 유에스 인크. Method and systems for prediction of hla class ii-specific epitopes and characterization of cd4+ t cells
CN113423419A (en) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 Methods and compositions for treating fabry disease
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
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
CN115023499A (en) 2019-08-16 2022-09-06 麻省理工学院 Targeted trans-splicing using CRISPR/Cas13
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
DE102020111571A1 (en) * 2020-03-11 2021-09-16 Immatics US, Inc. WPRE MUTANT CONSTRUCTS, COMPOSITIONS, AND RELATED PROCEDURES
WO2021181108A1 (en) * 2020-03-13 2021-09-16 Oxford Biomedica (Uk) Limited Lentiviral vectors
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
CA3195177A1 (en) 2020-10-07 2022-04-14 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy disorders including limb girdle 2i (lgmd2i)
JP2024501482A (en) 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 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
IL316038A (en) 2022-04-04 2024-11-01 Univ 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
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
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
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994024870A1 (en) * 1993-01-20 1994-11-10 Biotransplant, Inc. Retroviral vectors capable of expressing multimeric proteins from multiple translational initiation sites
US6103226A (en) * 1994-08-12 2000-08-15 Arch Development Corporation Genetically engineered cells that produce produce L. Dopa

Family Cites Families (32)

* 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
EP0854193A1 (en) 1990-09-21 1998-07-22 Chiron Corporation Human retroviral packaging cell line
ATE140966T1 (en) 1990-09-25 1996-08-15 Genentech Inc NEW NEUROTROPIC FACTOR
ATE213779T1 (en) 1991-08-07 2002-03-15 W French Anderson INTERNAL RIBOSOME ENTRY SITES CONTAIN RETROVIRAL VECTORS
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
AU3635695A (en) 1994-09-19 1996-04-09 Board Of Trustees Of The Leland Stanford Junior University Methods for genetically modifying hematopoietic stem cells
US5739307A (en) 1995-08-28 1998-04-14 Washington University Polynucleotide encoding neurturin neurotrophic factor
CA2234931C (en) 1995-10-16 2010-01-19 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
AU1073797A (en) 1995-11-14 1997-06-05 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
US6133027A (en) 1996-08-07 2000-10-17 City Of Hope Inducible expression system
US5739018A (en) 1996-08-07 1998-04-14 The Regents Of The University Of California Packaging cell lines for pseudotyped retroviral vectors
GB2322131B (en) * 1996-09-21 2000-12-20 Viromedica Pacific Ltd Improved retroviral vectors for gene therapy
PT904392E (en) * 1996-10-17 2001-06-29 Oxford Biomedica Ltd 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
IL132463A0 (en) * 1997-05-13 2001-03-19 Univ North Carolina Lentivirus - based gene transfer vectors
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
GB9720465D0 (en) 1997-09-25 1997-11-26 Oxford Biomedica Ltd Dual-virus vectors
KR20010052076A (en) * 1997-09-23 2001-06-25 찰스 굿맨 Expression of genes in hematopoietic stem cells in ischaemic conditions
GB2356200B (en) * 1997-12-22 2002-05-01 Oxford Biomedica Ltd Retroviral vectors
ES2373406T3 (en) 1997-12-22 2012-02-03 Oxford Biomedica (Uk) Limited VECTORS BASED ON THE VIRUS OF THE EQUINE INFECTIOUS ANEMIA (VAIE).
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
US6284540B1 (en) 1998-09-29 2001-09-04 Washington University Artemin, a novel neurotrophic factor
US6506378B1 (en) * 1998-12-16 2003-01-14 Arch Development Corporation Vesicular monoamine transporter gene therapy in Parkinson's disease

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994024870A1 (en) * 1993-01-20 1994-11-10 Biotransplant, Inc. Retroviral vectors capable of expressing multimeric proteins from multiple translational initiation sites
US6103226A (en) * 1994-08-12 2000-08-15 Arch Development Corporation Genetically engineered cells that produce produce L. Dopa

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MOFFAT M ET AL: "L-DOPA AND DOPAMINE-PRODUCING GENE CASSETTES FOR GENE THERAPY APPROACHES TO PARKINSON'S DISEASE", EXPERIMENTAL NEUROLOGY, SAN DIEGO, CA, US, vol. 144, no. 1, March 1997 (1997-03-01), pages 69 - 73, XP000901441 *
ROBBINS P D ET AL: "Viral vectors for gene therapy", TRENDS IN BIOTECHNOLOGY, ELSEVIER PUBLICATIONS, CAMBRIDGE, GB, vol. 16, no. 1, 1998, pages 35 - 40, XP004101894, ISSN: 0167-7799 *

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