GB0024550D0 - - Google Patents

Info

Publication number
GB0024550D0
GB0024550D0 GBGB0024550.6A GB0024550A GB0024550D0 GB 0024550 D0 GB0024550 D0 GB 0024550D0 GB 0024550 A GB0024550 A GB 0024550A GB 0024550 D0 GB0024550 D0 GB 0024550D0
Authority
GB
United Kingdom
Prior art keywords
vector genome
genomes
relates
present
nois
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
GBGB0024550.6A
Other languages
English (en)
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
Priority to GBGB0024550.6A priority Critical patent/GB0024550D0/en
Publication of GB0024550D0 publication Critical patent/GB0024550D0/en
Priority to ES01972317T priority patent/ES2348277T3/es
Priority to PCT/GB2001/004433 priority patent/WO2002029065A2/en
Priority to PT01972317T priority patent/PT1337655E/pt
Priority to CA002424738A priority patent/CA2424738A1/en
Priority to EP01972317A priority patent/EP1337655B1/en
Priority to JP2002532634A priority patent/JP4224295B2/ja
Priority to DK01972317.0T priority patent/DK1337655T3/da
Priority to AU2001292093A priority patent/AU2001292093A1/en
Priority to EP09014156A priority patent/EP2180057A1/en
Priority to DE60142576T priority patent/DE60142576D1/de
Priority to AT01972317T priority patent/ATE474058T1/de
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
Priority to CY20101100888T priority patent/CY1110817T1/el
Ceased legal-status Critical Current

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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (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)
  • Molds, Cores, And Manufacturing Methods Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
GBGB0024550.6A 2000-10-06 2000-10-06 Ceased GB0024550D0 (enExample)

Priority Applications (17)

Application Number Priority Date Filing Date Title
GBGB0024550.6A GB0024550D0 (enExample) 2000-10-06 2000-10-06
AT01972317T ATE474058T1 (de) 2000-10-06 2001-10-05 Lentivirale vektoren zur behandlung neurodegenerativer krankheiten
AU2001292093A AU2001292093A1 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites
DE60142576T DE60142576D1 (de) 2000-10-06 2001-10-05 Lentivirale vektoren zur behandlung neurodegenerativer krankheiten
PT01972317T PT1337655E (pt) 2000-10-06 2001-10-05 Vectores lentivirais para o tratamento de doenças neurodegenerativas
CA002424738A CA2424738A1 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites
EP01972317A EP1337655B1 (en) 2000-10-06 2001-10-05 Lentiviral vectors for the treatment of neurodegenerative diseases
JP2002532634A JP4224295B2 (ja) 2000-10-06 2001-10-05 ベクターシステム
DK01972317.0T DK1337655T3 (da) 2000-10-06 2001-10-05 Lentivirusvektorer til behandling af neurodegenerative sygdomme
ES01972317T ES2348277T3 (es) 2000-10-06 2001-10-05 Vectores lentivíricos para el tratamiento de las enfermedades neurodegenerativas.
EP09014156A EP2180057A1 (en) 2000-10-06 2001-10-05 Vector system
PCT/GB2001/004433 WO2002029065A2 (en) 2000-10-06 2001-10-05 Retroviral vectors containing internal ribosomal entry sites
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
CY20101100888T CY1110817T1 (el) 2000-10-06 2010-10-06 Βραδεων ιων φορεις για τη θεραπεια νευροεκφυλιστικων ασθενειων

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
GB0024550D0 true GB0024550D0 (enExample) 2000-11-22

Family

ID=9900826

Family Applications (1)

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

Country Status (13)

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

Families Citing this family (134)

* 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
EP2348119B1 (en) * 2002-02-01 2017-04-26 Oxford BioMedica (UK) Limited Multicistronic lentiviral vector
JP5519287B2 (ja) * 2006-11-02 2014-06-11 ダニエル・ジェイ・カポン 可動部を備えたハイブリッド免疫グロブリン
EP2307551B1 (en) 2008-06-18 2016-12-14 Oxford BioMedica (UK) Limited Purification of retroviral vectors
US8829173B2 (en) * 2008-09-26 2014-09-09 Tocagen Inc. Recombinant vectors
WO2010055290A1 (en) * 2008-11-11 2010-05-20 Oxford Biomedica (Uk) Limited Method
US20120309816A1 (en) 2009-11-09 2012-12-06 Genepod Therapeutics Ab Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
EP2575894B1 (en) * 2010-05-28 2015-02-25 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
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
JP6698114B2 (ja) * 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 構築物
GB201118636D0 (en) * 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
US10400252B2 (en) 2011-10-28 2019-09-03 Oxford Biomedica (Uk) Ltd. Catecholamine enzyme fusions
US9347065B2 (en) 2012-03-29 2016-05-24 International Aids Vaccine Initiative Methods to improve vector expression and genetic stability
JP6279466B2 (ja) 2012-04-27 2018-02-14 Ｊｃｒファーマ株式会社 新規な発現ベクター
WO2014043131A1 (en) * 2012-09-14 2014-03-20 The Regents Of The University Of 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
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, 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
WO2014093965A1 (en) * 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
EP3011031B1 (en) 2013-06-17 2020-09-30 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
KR20160056869A (ko) 2013-06-17 2016-05-20 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
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
KR20250068794A (ko) 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CN106103705A (zh) 2013-12-12 2016-11-09 布罗德研究所有限公司 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法
CA2932479A1 (en) 2013-12-12 2015-06-18 The Rockefeller University Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
EP3082853A2 (en) 2013-12-20 2016-10-26 The Broad Institute, Inc. 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
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
WO2016094867A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
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)
ES3049406T3 (en) 2014-12-19 2025-12-16 Broad Inst Inc Methods for profiling the t-cell-receptor repertoire
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
EP3234193B1 (en) 2014-12-19 2020-07-15 Massachusetts Institute of Technology Molecular biomarkers for cancer immunotherapy
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
ES2578903B1 (es) * 2015-01-02 2017-07-07 Instituto De Salud Carlos Iii Vector lentiviral de expresión autolimitada
US20180271891A1 (en) 2015-03-11 2018-09-27 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
CN108025048B (zh) 2015-05-20 2022-10-25 博德研究所 共有的新抗原
EP3822291A1 (en) 2015-06-10 2021-05-19 The Broad Institute Inc. Antibodies, compounds and screens for identifying and treating cachexia or pre-cachexia
FI3430134T3 (fi) 2015-06-18 2023-01-13 Uusia CRISPR-entsyymejä ja järjestelmiä
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
WO2017021359A1 (en) * 2015-08-03 2017-02-09 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
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
KR20250021623A (ko) 2015-10-22 2025-02-13 더 브로드 인스티튜트, 인코퍼레이티드 타입 vi-b crispr 효소 및 시스템
WO2017074788A1 (en) 2015-10-27 2017-05-04 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
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
EP3446119A1 (en) 2016-04-18 2019-02-27 The Broad Institute Inc. Improved hla epitope prediction
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
US12410416B2 (en) 2016-04-19 2025-09-09 The Broad Institute, Inc. CRISPR enzymes and systems with modified pam specificity
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
PL3445406T3 (pl) * 2016-04-20 2022-02-07 Centro De Investigaciones Energéticas, Medioambientales Y Tecnológicas, O.A., M.P. Kompozycje i sposoby zwiększonej ekspresji genów PKLR
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2017219027A1 (en) 2016-06-17 2017-12-21 The Broad Institute Inc. Type vi crispr orthologs and systems
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
CN109804246B (zh) * 2016-08-01 2022-05-17 健康研究公司 用于快速克隆t细胞受体的组合物和方法
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
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 (ko) * 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료
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
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP4667565A2 (en) 2016-10-20 2025-12-24 Sangamo Therapeutics, Inc. 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
SI3580561T1 (sl) 2017-02-12 2024-04-30 Biontech Us Inc. Metode, osnovane na hla, in njihove sestave ter uporabe
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
KR20240155360A (ko) 2017-04-12 2024-10-28 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
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
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3648781A4 (en) 2017-07-07 2021-05-19 The Broad Institute, Inc. Crispr system based antiviral therapy
CA3073848A1 (en) 2017-09-21 2019-03-28 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
EP3692152B1 (en) 2017-10-04 2025-09-03 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
US12018080B2 (en) 2017-11-13 2024-06-25 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the CLEC2D-KLRB1 pathway
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
EP3728575A4 (en) 2017-12-22 2021-11-24 The Broad Institute, Inc. CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF DNA BASES
JP7197901B2 (ja) * 2018-01-11 2022-12-28 学校法人慈恵大学 Ｈｕｎｔｅｒ症候群の遺伝子治療に使用されるベクタープラスミド、レンチウイルスベクターシステム、細胞、及び、細胞製剤
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
RU2020134965A (ru) 2018-04-27 2022-04-27 Спейскрафт Севен, Ллк Генная терапия при дегенерации цнс
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN116024271A (zh) * 2018-05-31 2023-04-28 康霖生物科技（杭州）有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
WO2020033601A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Novel 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
US20220062394A1 (en) 2018-12-17 2022-03-03 The Broad Institute, Inc. Methods for identifying neoantigens
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
PH12021551485A1 (en) 2018-12-21 2022-04-11 Biontech Us Inc Method and systems for prediction of hla class ii-specific epitopes and characterization of cd4+ t cells
KR20210112337A (ko) * 2019-01-04 2021-09-14 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
EP3937985A1 (en) 2019-03-10 2022-01-19 Sio Gene Therapies Inc. Gene therapy compositions and methods for treating parkinson's disease
US20220177863A1 (en) 2019-03-18 2022-06-09 The Broad Institute, Inc. Type vii crispr proteins and systems
US20220220469A1 (en) 2019-05-20 2022-07-14 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
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 (de) * 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
EP4118217A1 (en) * 2020-03-13 2023-01-18 Oxford BioMedica (UK) Limited Lentiviral vectors
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
EP4225382A4 (en) 2020-10-07 2025-04-23 AskBio Inc. ADMINISTRATION OF THERAPEUTIC ADENO-ASSOCIATED VIRUS FUKKUTIN-ASSOCIATED PROTEIN (FKRP) FOR THE TREATMENT OF DYSTROGLYCANOPATHY. DISORDERS INCLUDING LIMB GIRDLE 21
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
CN114107396B (zh) * 2021-11-26 2024-02-02 和元智造(上海)基因技术有限公司 慢病毒载体、系统及其应用
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
WO2024010067A1 (ja) * 2022-07-08 2024-01-11 Ｊｃｒファーマ株式会社 中枢神経系疾患の治療のための、核酸分子、ベクター、組換え細胞及び薬剤
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
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
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

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
AU6075190A (en) 1989-06-30 1991-01-17 Regents Of The University Of California, The Retrovirus detection
AU665176B2 (en) 1990-09-21 1995-12-21 Novartis Vaccines And Diagnostics, Inc. Packaging cells
DK0550665T3 (da) 1990-09-25 1996-12-02 Genentech Inc Ny neurotrofisk faktor
DE69232438T2 (de) 1991-08-07 2002-10-10 W. French Anderson Interne ribosom eintrittsstellen enthaltene retrovirale vektoren
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
WO1997014809A2 (en) 1995-10-16 1997-04-24 Dana-Farber Cancer Institute Novel expression vectors and methods of use
EP0859856A2 (en) 1995-11-08 1998-08-26 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
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
US6190907B1 (en) * 1996-09-21 2001-02-20 Viromedica Pacific Limited Retroviral vectors for gene therapy
CN1195863C (zh) 1996-10-17 2005-04-06 牛津生物医学（英国）有限公司 逆转录病毒载体
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
KR100556864B1 (ko) * 1997-05-13 2006-03-10 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 렌티바이러스-기원 유전자 전달 벡터
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
EP1017838A2 (en) * 1997-09-23 2000-07-12 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
GB2356200B (en) * 1997-12-22 2002-05-01 Oxford Biomedica Ltd Retroviral vectors
CN1322137C (zh) 1997-12-22 2007-06-20 牛津生物医学（英国）有限公司 基于马传染性贫血病毒(eiav)的逆转录病毒载体
GB9803351D0 (en) 1998-02-17 1998-04-15 Oxford Biomedica Ltd Anti-viral vectors
GB2351290A (en) 1998-05-22 2000-12-27 Oxford Biomedica Ltd Retroviral delivery sytem
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

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