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
Other languages
English (en)
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

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)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Neurosurgery (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (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)
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
GB0024550.6 2000-10-06
GBGB0024550.6A GB0024550D0 (enrdf_load_stackoverflow) 2000-10-06 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 (enrdf_load_stackoverflow)
EP (2) EP1337655B1 (enrdf_load_stackoverflow)
JP (1) JP4224295B2 (enrdf_load_stackoverflow)
AT (1) ATE474058T1 (enrdf_load_stackoverflow)
AU (1) AU2001292093A1 (enrdf_load_stackoverflow)
CA (1) CA2424738A1 (enrdf_load_stackoverflow)
CY (1) CY1110817T1 (enrdf_load_stackoverflow)
DE (1) DE60142576D1 (enrdf_load_stackoverflow)
DK (1) DK1337655T3 (enrdf_load_stackoverflow)
ES (1) ES2348277T3 (enrdf_load_stackoverflow)
GB (1) GB0024550D0 (enrdf_load_stackoverflow)
PT (1) PT1337655E (enrdf_load_stackoverflow)
WO (1) WO2002029065A2 (enrdf_load_stackoverflow)

Families Citing this family (130)

* 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
ES2411981T3 (es) * 2002-02-01 2013-07-09 Oxford Biomedica (Uk) Limited Vector lentivírico
EP2078040B1 (en) * 2006-11-02 2017-06-28 Daniel J. Capon Methods of producing hybrid polypeptides with moving parts
JP5615271B2 (ja) 2008-06-18 2014-10-29 オックスフォード バイオメディカ（ユーケー）リミテッド ウイルス精製法
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
CN102695526A (zh) * 2009-11-09 2012-09-26 吉恩波多治疗股份公司 用于神经元特异性的体内连续dopa合成的新型病毒载体构建体
JP5965392B2 (ja) * 2010-05-28 2016-08-03 オックスフォード バイオメディカ （ユーケー） リミテッド 脳へのレンチウイルスベクターの送達
WO2012135389A2 (en) * 2011-03-28 2012-10-04 The Regents Of The University Of California Host cells and methods for oxidizing aromatic amino acids
US20130189754A1 (en) 2011-09-12 2013-07-25 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
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
EP2848687B1 (en) 2012-04-27 2017-09-13 JCR Pharmaceuticals Co., Ltd. Novel expression vector
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
US11065311B2 (en) 2012-10-25 2021-07-20 Denovo Biopharma Llc Retroviral vector with mini-promoter cassette
SG11201504523UA (en) 2012-12-12 2015-07-30 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
WO2014093965A1 (en) 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
WO2015048690A1 (en) * 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
US10801070B2 (en) 2013-11-25 2020-10-13 The Broad Institute, Inc. Compositions and methods for diagnosing, evaluating and treating cancer
US11725237B2 (en) 2013-12-05 2023-08-15 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
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
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Ｈｂｖ 및 바이러스 질병 및 질환을 위한 ｃｒｉｓｐｒ­ｃａｓ 시스템 및 조성물의 전달，용도 및 치료적 적용
CN106061510B (zh) 2013-12-12 2020-02-14 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
US11452768B2 (en) 2013-12-20 2022-09-27 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
EP3212788A2 (en) 2014-10-27 2017-09-06 The Broad Institute, Inc. Compositions, methods and use of synthetic lethal screening
JP6401871B2 (ja) 2014-11-05 2018-10-10 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
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
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
WO2016100975A1 (en) 2014-12-19 2016-06-23 Massachsetts Institute Ot Technology Molecular biomarkers for cancer immunotherapy
EP3234130B1 (en) 2014-12-19 2020-11-25 The Broad Institute, Inc. Methods for profiling the t-cell- receptor repertoire
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 (es) * 2015-01-02 2017-07-07 Instituto De Salud Carlos Iii Vector lentiviral de expresión autolimitada
EP3268044A2 (en) 2015-03-11 2018-01-17 The Broad Institute Inc. Prmt5 inhibitors for the treatment of cancer with reduced mtap activty
JP6913032B2 (ja) 2015-05-20 2021-08-04 ザ・ブロード・インスティテュート・インコーポレイテッド 共通ネオ抗原
US20180355033A1 (en) 2015-06-10 2018-12-13 Dana-Farber Cancer Institute, Inc. Antibodies, compounds and screens for identifying and treating cachexia or pre-cachexia
CN108290933A (zh) 2015-06-18 2018-07-17 布罗德研究所有限公司 降低脱靶效应的crispr酶突变
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
JP2018522595A (ja) * 2015-08-03 2018-08-16 ミョドパ・リミテッド Ｌ−ｄｏｐａの全身合成及び調節
US11214800B2 (en) 2015-08-18 2022-01-04 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
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
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
WO2017106657A1 (en) 2015-12-18 2017-06-22 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
CA3026112A1 (en) 2016-04-19 2017-10-26 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
EP3445848A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017189308A1 (en) 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems
DK3445406T3 (da) * 2016-04-20 2021-08-30 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Sammensætninger og fremgangsmåder til forøget genekspression af pklr
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
KR20190019168A (ko) 2016-06-17 2019-02-26 더 브로드 인스티튜트, 인코퍼레이티드 제vi형 crispr 오솔로그 및 시스템
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
WO2018026827A1 (en) * 2016-08-01 2018-02-08 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
EP3500671B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method of selecting target sequences for the design of guide rnas
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
CN110536966A (zh) * 2016-09-08 2019-12-03 能源环境和技术研究中心O.A.,M.P. 用于范可尼贫血患者的基因疗法
US20200016202A1 (en) 2016-10-07 2020-01-16 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
EP3528852B1 (en) 2016-10-20 2025-09-03 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
EP3574116A1 (en) 2017-01-24 2019-12-04 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
ES2950676T3 (es) 2017-01-30 2023-10-11 Kws Saat Se & Co Kgaa Reparación de enlace de plantillas a endonucleasas para modificación de genes
CN116693695A (zh) 2017-02-12 2023-09-05 百欧恩泰美国公司 基于hla的方法和组合物及其用途
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
US11840711B2 (en) 2017-04-12 2023-12-12 The Broad Institute, Inc. Type VI CRISPR orthologs and systems
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. 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 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111511388A (zh) 2017-09-21 2020-08-07 博德研究所 用于靶向核酸编辑的系统、方法和组合物
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
EP3697452A4 (en) 2017-10-16 2021-11-24 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A., M.P. LENTIVIRAL VECTORS FOR THE ADMINISTRATION 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
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
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
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
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
CN108841868A (zh) * 2018-05-31 2018-11-20 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
US20210163944A1 (en) 2018-08-07 2021-06-03 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
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
US11384344B2 (en) 2018-12-17 2022-07-12 The Broad Institute, Inc. CRISPR-associated transposase systems and methods of use thereof
JP7236543B2 (ja) 2018-12-21 2023-03-09 ビオンテック ユーエス インコーポレイテッド Ｈｌａクラスｉｉ特異的エピトープの予測およびｃｄ４＋ ｔ細胞の特徴付けのための方法およびシステム
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
CN115835887A (zh) 2019-03-10 2023-03-21 牛津生物医学（英国）有限公司 用于治疗帕金森病的基因治疗组合物和方法
WO2020191102A1 (en) 2019-03-18 2020-09-24 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
JP2022544688A (ja) 2019-08-16 2022-10-20 マサチューセッツ インスティテュート オブ テクノロジー Ｃｒｉｓｐｒ／ｃａｓ１３を使用する標的化トランススプライシング
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
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
WO2022076556A2 (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)
WO2022132596A2 (en) 2020-12-14 2022-06-23 Biontech Us Inc. Tissue-specific antigens for cancer immunotherapy
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
EP4503923A1 (en) 2022-04-04 2025-02-12 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

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 (ja) 1990-09-21 1994-01-27 カイロン　コーポレイション パッケージング細胞
JPH06501617A (ja) 1990-09-25 1994-02-24 ジェネンテク，インコーポレイテッド 新規な神経栄養因子
EP0598029B1 (en) 1991-08-07 2002-02-27 ANDERSON, W., French Retroviral vectors containing internal ribosome entry sites
JPH08510636A (ja) * 1993-01-20 1996-11-12 バイオトランスプラント，インコーポレイテッド 多重翻訳開始部位から多量体タンパク質を発現出来るレトロウイルスベクター
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
AU7435296A (en) 1995-10-16 1997-05-07 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
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
WO1998012338A1 (en) 1996-09-21 1998-03-26 Viromedica Pacific Limited Improved retroviral vectors for gene therapy
ATE198910T1 (de) * 1996-10-17 2001-02-15 Oxford Biomedica Ltd Retrovirale vektoren
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
JP4390860B2 (ja) * 1997-05-13 2009-12-24 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル レンチウイルスをベースにした遺伝子転移ベクター
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
WO1999015684A2 (en) * 1997-09-23 1999-04-01 Oxford Biomedica (Uk) Limited Expression of genes in hematopoietic stem cells in hischaemic conditions
DK1895010T3 (da) 1997-12-22 2011-11-21 Oxford Biomedica Ltd Vektorer baseret på virus for infektiøs hesteanæmi (eiav)
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
CN1325653C (zh) 1998-05-22 2007-07-11 牛津生物医学（英国）有限公司 逆转录病毒传递系统
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

Also Published As

Publication number Publication date
ES2348277T3 (es) 2010-12-02
GB0024550D0 (enrdf_load_stackoverflow) 2000-11-22
CA2424738A1 (en) 2002-04-11
US20070025970A1 (en) 2007-02-01
US20090111106A1 (en) 2009-04-30
PT1337655E (pt) 2010-08-25
EP1337655A2 (en) 2003-08-27
DK1337655T3 (da) 2010-09-20
DE60142576D1 (de) 2010-08-26
EP1337655B1 (en) 2010-07-14
US20040013648A1 (en) 2004-01-22
JP4224295B2 (ja) 2009-02-12
CY1110817T1 (el) 2015-06-10
WO2002029065A3 (en) 2002-12-27
ATE474058T1 (de) 2010-07-15
US7259015B2 (en) 2007-08-21
WO2002029065A2 (en) 2002-04-11
JP2004520016A (ja) 2004-07-08
EP2180057A1 (en) 2010-04-28

Similar Documents

Publication Publication Date Title
AU2001292093A1 (en) Retroviral vectors containing internal ribosomal entry sites
DK1351703T3 (da) Anvendelse af et polypeptid omfattende det ekstracellulære domæne af IL-20RA og IL-20RB til behandling af inflammation
AU2001252615A2 (en) Method for attaching authentication bar code, authentication method, apparatus for attaching authentication bar code, authentication apparatus and portable terminal
AU2001252615A1 (en) Method for attaching authentication bar code, authentication method, apparatus for attaching authentication bar code, authentication apparatus and portable terminal
WO2003008444A3 (en) Peptides effective in the treatment of tumors and other conditions requiring the removal or destruction of cells
EP1457558A8 (en) Weed controller metabolism proteins, genes thereof and use of the same
WO2005058959A3 (en) Treatment of viral infections
NO20011017D0 (no) Polymeriserings-inhibitering av styren-monomerer
MXPA04012105A (es) Metodo, sistema yterminal para recibir el contenido con acceso autorizado.
WO2002065993A3 (en) Use of aryl nitrone compounds in methods for treating neuropathic pain
CA2296560A1 (en) Amino acid derivatives useful to treat stroke
AU2001248822A1 (en) Gd3-mimetic peptides
WO2003040290A3 (de) Gene aus corynebacterium glutamicum die fuer homeostase- und adaptions-proteine codieren
AUPP627498A0 (en) Novel peptides - i
ZA989705B (en) Epoxide hydrolase
EP1762612A3 (en) Insertion sequence elements derived from ralstonia solanacearum
ATE412751T1 (de) Menschliche tak1 und für diese kodierende dna
WO2001048185A3 (en) Transcription regulatory sequences derived from chlamydomonas reinhardtii
DK1425295T3 (da) Modificerede peptider og deres anvendelse til behandling af autoimmune sygdomme
WO2002046378A3 (en) Alternative pol k nucleotide and amino acid sequence and methods for using
WO2003041640A3 (en) METHODS FOR TREATING ISCHEMIC REPERFUSION INJURY USING IkB KINASE-BETA INHIBITORS
Rösch Endoscopic Ultrasonography-More Questions than Answers?
DK1280890T3 (da) Mutante stammer, der er i stand til at producere kemisk differentierede proteiner ved inkorporering af ikke-traditionelle aminosyrer
WO2004003170A3 (en) Agents capable of inhibiting ras and uses thereof
WO2001092490A3 (en) Isolated human aminotransferase proteins, nucleic acid molecules encoding human aminotransferase proteins, and uses thereof