DK1337655T3 - Lentivirusvektorer til behandling af neurodegenerative sygdomme - Google Patents
Lentivirusvektorer til behandling af neurodegenerative sygdommeInfo
- Publication number
- DK1337655T3 DK1337655T3 DK01972317.0T DK01972317T DK1337655T3 DK 1337655 T3 DK1337655 T3 DK 1337655T3 DK 01972317 T DK01972317 T DK 01972317T DK 1337655 T3 DK1337655 T3 DK 1337655T3
- Authority
- DK
- Denmark
- Prior art keywords
- treatment
- neurodegenerative diseases
- vector genome
- lentivirus vectors
- genomes
- Prior art date
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)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (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)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GBGB0024550.6A GB0024550D0 (da) | 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 |
---|---|
DK1337655T3 true DK1337655T3 (da) | 2010-09-20 |
Family
ID=9900826
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK01972317.0T DK1337655T3 (da) | 2000-10-06 | 2001-10-05 | Lentivirusvektorer til behandling af neurodegenerative sygdomme |
Country Status (13)
Country | Link |
---|---|
US (3) | US7259015B2 (da) |
EP (2) | EP1337655B1 (da) |
JP (1) | JP4224295B2 (da) |
AT (1) | ATE474058T1 (da) |
AU (1) | AU2001292093A1 (da) |
CA (1) | CA2424738A1 (da) |
CY (1) | CY1110817T1 (da) |
DE (1) | DE60142576D1 (da) |
DK (1) | DK1337655T3 (da) |
ES (1) | ES2348277T3 (da) |
GB (1) | GB0024550D0 (da) |
PT (1) | PT1337655E (da) |
WO (1) | WO2002029065A2 (da) |
Families Citing this family (116)
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 |
ES2634424T3 (es) * | 2002-02-01 | 2017-09-27 | Oxford Biomedica (Uk) Limited | Vector multicistrónico lentivírico |
AU2007353396C1 (en) * | 2006-11-02 | 2013-06-27 | Biomolecular Holdings Llc | Hybrid immunoglobulins with moving parts |
EP3192874B1 (en) | 2008-06-18 | 2019-10-16 | Oxford BioMedica (UK) Limited | Virus purification |
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 |
NZ600104A (en) * | 2009-11-09 | 2013-11-29 | Genepod Therapeutics Ab | Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo |
CN102946907A (zh) * | 2010-05-28 | 2013-02-27 | 牛津生物医学(英国)有限公司 | 慢病毒载体向脑的递送 |
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 |
US10400252B2 (en) | 2011-10-28 | 2019-09-03 | Oxford Biomedica (Uk) Ltd. | Catecholamine enzyme fusions |
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 |
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 |
JP2015529466A (ja) * | 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 | 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター |
CN104884627A (zh) | 2012-10-25 | 2015-09-02 | 托卡根公司 | 具有小型启动子盒的逆转录病毒载体 |
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 |
MX2015007550A (es) | 2012-12-12 | 2017-02-02 | Broad Inst Inc | Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas. |
WO2014093965A1 (en) * | 2012-12-14 | 2014-06-19 | Case Western Reserve University | Genomic rna packaging enhancer element |
AU2014281028B2 (en) | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
JP6702858B2 (ja) | 2013-06-17 | 2020-06-03 | ザ・ブロード・インスティテュート・インコーポレイテッド | ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用 |
CA2915845A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
US10822606B2 (en) | 2013-09-27 | 2020-11-03 | 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 |
EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
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 |
SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
CN111206032A (zh) | 2013-12-12 | 2020-05-29 | 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 |
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 |
KR20160101073A (ko) | 2013-12-20 | 2016-08-24 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신생항원 백신과의 병용 요법 |
WO2016028682A1 (en) | 2014-08-17 | 2016-02-25 | The Broad Institute Inc. | Genome editing using cas9 nickases |
WO2016049163A2 (en) | 2014-09-24 | 2016-03-31 | The Broad Institute Inc. | Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder |
WO2016049258A2 (en) | 2014-09-25 | 2016-03-31 | The Broad Institute Inc. | Functional screening with optimized functional crispr-cas systems |
EP3212788A2 (en) | 2014-10-27 | 2017-09-06 | The Broad Institute, Inc. | Compositions, methods and use of synthetic lethal screening |
BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
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) |
WO2016094867A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Protected guide rnas (pgrnas) |
WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
WO2016100977A1 (en) | 2014-12-19 | 2016-06-23 | The Broad Institute Inc. | Methods for profiling the t-cel- receptor repertoire |
WO2016100975A1 (en) | 2014-12-19 | 2016-06-23 | Massachsetts Institute Ot Technology | Molecular biomarkers for cancer immunotherapy |
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 |
WO2016106236A1 (en) | 2014-12-23 | 2016-06-30 | The Broad Institute Inc. | Rna-targeting system |
AU2015369725A1 (en) | 2014-12-24 | 2017-06-29 | 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 |
CN116196401A (zh) | 2015-05-20 | 2023-06-02 | 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 |
AU2016279062A1 (en) | 2015-06-18 | 2019-03-28 | Omar O. Abudayyeh | Novel CRISPR enzymes and systems |
EP3666895A1 (en) | 2015-06-18 | 2020-06-17 | 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 |
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 |
WO2017021359A1 (en) * | 2015-08-03 | 2017-02-09 | Myodopa Limited | Systemic synthesis and regulation of l-dopa |
EP3337908A4 (en) | 2015-08-18 | 2019-01-23 | The Broad Institute, Inc. | METHOD AND COMPOSITIONS FOR CHANGING THE FUNCTION AND STRUCTURE OF CHROMATIN GRINDING AND / OR DOMAINS |
WO2017069958A2 (en) | 2015-10-09 | 2017-04-27 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
KR20180133374A (ko) | 2015-10-22 | 2018-12-14 | 더 브로드 인스티튜트, 인코퍼레이티드 | 타입 vi-b crispr 효소 및 시스템 |
EP3368687B1 (en) | 2015-10-27 | 2021-09-29 | The Broad Institute, Inc. | Compositions and methods for targeting cancer-specific sequence variations |
WO2017075451A1 (en) | 2015-10-28 | 2017-05-04 | The Broad Institute Inc. | Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1 |
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 |
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 |
EP3443001A4 (en) | 2016-04-11 | 2020-04-29 | Obsidian Therapeutics, Inc. | REGULATED BIOCIRCUIT SYSTEMS |
AU2017254477A1 (en) | 2016-04-18 | 2018-11-01 | Jennifer G. ABELIN | Improved HLA epitope prediction |
US11286478B2 (en) | 2016-04-19 | 2022-03-29 | The Broad Institute, Inc. | Cpf1 complexes with reduced indel activity |
AU2017257274B2 (en) | 2016-04-19 | 2023-07-13 | Massachusetts Institute Of Technology | Novel CRISPR enzymes and systems |
KR102670601B1 (ko) | 2016-04-19 | 2024-05-29 | 더 브로드 인스티튜트, 인코퍼레이티드 | 신규한 crispr 효소 및 시스템 |
BR112018071695A2 (pt) | 2016-04-20 | 2019-02-19 | Centro De Investigaciones Energeticas, Medioambientales Y Tecnologicas | composições e métodos para a expressão gênica acentuada de pklr |
JP7267013B2 (ja) | 2016-06-17 | 2023-05-01 | ザ・ブロード・インスティテュート・インコーポレイテッド | Vi型crisprオルソログ及び系 |
WO2018005873A1 (en) | 2016-06-29 | 2018-01-04 | The Broad Institute Inc. | Crispr-cas systems having destabilization domain |
US20190183935A1 (en) * | 2016-08-01 | 2019-06-20 | Health Research, Inc. | Compositions and methods for rapid cloning of t-cell receptors |
CN110114461A (zh) | 2016-08-17 | 2019-08-09 | 博德研究所 | 新型crispr酶和系统 |
WO2018035387A1 (en) | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
US20190262399A1 (en) | 2016-09-07 | 2019-08-29 | The Broad Institute, Inc. | Compositions and methods for evaluating and modulating immune responses |
JP7197466B2 (ja) * | 2016-09-08 | 2022-12-27 | セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー.エイ. エム.ピー. | ファンコニ貧血患者に対する遺伝子療法 |
WO2018067991A1 (en) | 2016-10-07 | 2018-04-12 | The Brigham And Women's Hospital, Inc. | Modulation of novel immune checkpoint targets |
CN110022904B (zh) | 2016-10-20 | 2024-04-19 | 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 |
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 |
FI3580561T3 (fi) | 2017-02-12 | 2023-12-12 | Biontech Us Inc | Hla-pohjaiset menetelmät ja koostumukset sekä niiden käyttö |
CA3056236A1 (en) | 2017-03-15 | 2018-09-20 | The Broad Institute, Inc. | Novel cas13b orthologues crispr enzymes and systems |
WO2018191388A1 (en) | 2017-04-12 | 2018-10-18 | The Broad Institute, Inc. | Novel type vi crispr orthologs and systems |
WO2018191750A2 (en) | 2017-04-14 | 2018-10-18 | The Broad Institute Inc. | Novel delivery of large payloads |
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 |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
AU2018338318B2 (en) | 2017-09-21 | 2022-12-22 | Massachusetts Institute Of Technology | Systems, methods, and compositions for targeted nucleic acid editing |
US20200255828A1 (en) | 2017-10-04 | 2020-08-13 | The Broad Institute, Inc. | Methods and compositions for altering function and structure of chromatin loops and/or domains |
JP7403461B2 (ja) | 2017-10-16 | 2023-12-22 | セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー.エイ. エム.ピー. | ピルビン酸キナーゼ欠損症を治療するためのpklr送達用レンチウイルスベクター |
US11547614B2 (en) | 2017-10-31 | 2023-01-10 | The Broad Institute, Inc. | Methods and compositions for studying cell evolution |
EP3710039A4 (en) | 2017-11-13 | 2021-08-04 | The Broad Institute, Inc. | METHODS AND COMPOSITIONS FOR CANCER TREATMENT BY TARGETING THE CLEC2D-KLRB1 PATH |
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 |
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 |
WO2020033601A1 (en) | 2018-08-07 | 2020-02-13 | The Broad Institute, Inc. | Novel cas12b enzymes and systems |
US20210317429A1 (en) | 2018-08-20 | 2021-10-14 | The Broad Institute, Inc. | Methods and compositions for optochemical control of crispr-cas9 |
US20210382068A1 (en) | 2018-10-02 | 2021-12-09 | Dana-Farber Cancer Institute, Inc. | Hla single allele lines |
WO2020131862A1 (en) | 2018-12-17 | 2020-06-25 | The Broad Institute, Inc. | Crispr-associated transposase systems and methods of use thereof |
WO2020131586A2 (en) | 2018-12-17 | 2020-06-25 | The Broad Institute, Inc. | Methods for identifying neoantigens |
AU2019404547A1 (en) | 2018-12-21 | 2021-07-22 | Biontech Us Inc. | Method and systems for prediction of HLA class II-specific epitopes and characterization of CD4+ T cells |
MX2021008131A (es) * | 2019-01-04 | 2021-10-13 | Sangamo Therapeutics Inc | Metodos y composiciones para el tratamiento de la enfermedad de fabry. |
CA3133188A1 (en) | 2019-03-10 | 2020-09-17 | Oxford Biomedica (Uk) Limited | 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 |
WO2020236972A2 (en) | 2019-05-20 | 2020-11-26 | The Broad Institute, Inc. | Non-class i multi-component nucleic acid targeting systems |
WO2021034717A1 (en) | 2019-08-16 | 2021-02-25 | Massachusetts Institute Of Technology | Targeted trans-splicing using crispr/cas13 |
MX2023001615A (es) | 2020-08-07 | 2023-03-08 | Spacecraft Seven Llc | Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. |
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) |
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 |
WO2023196818A1 (en) | 2022-04-04 | 2023-10-12 | 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 |
Family Cites Families (34)
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 |
WO1992005266A2 (en) | 1990-09-21 | 1992-04-02 | Viagene, Inc. | Packaging cells |
EP0550665B1 (en) | 1990-09-25 | 1996-07-31 | Genentech, Inc. | Novel neurothrophic factor |
CA2114416C (en) | 1991-08-07 | 1998-07-07 | W. French Anderson | Retroviral vectors containing internal ribosome entry sites |
CA2162056A1 (en) * | 1993-01-20 | 1994-11-10 | Christian A. Le Guern | Retroviral vectors capable of expressing multimeric proteins from multiple translational initiation sites |
US5834256A (en) | 1993-06-11 | 1998-11-10 | Cell Genesys, Inc. | Method for production of high titer virus and high efficiency retroviral mediated transduction of mammalian cells |
US5728803A (en) | 1994-06-03 | 1998-03-17 | Genentech, Inc. | Pantropic neurotrophic factors |
US6103226A (en) * | 1994-08-12 | 2000-08-15 | Arch Development Corporation | Genetically engineered cells that produce produce L. Dopa |
WO1996009400A1 (en) | 1994-09-19 | 1996-03-28 | Systemix, Inc. | Methods for genetically modifying hematopoietic stem cells |
US5739307A (en) | 1995-08-28 | 1998-04-14 | Washington University | Polynucleotide encoding neurturin neurotrophic factor |
EP0857217B1 (en) | 1995-10-16 | 2005-04-13 | Dana-Farber Cancer Institute | Novel expression vectors and methods of use |
WO1997017457A2 (en) | 1995-11-08 | 1997-05-15 | 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 |
KR100246096B1 (ko) * | 1996-09-21 | 2000-03-15 | 이선경 | 유전자 요법을 위한 개선된 레트로바이러스 벡터 |
DE69703974T2 (de) * | 1996-10-17 | 2001-07-19 | 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 |
NZ503318A (en) * | 1997-09-23 | 2001-11-30 | Oxford Biomedica Ltd | Modified haemopoietic stem cell comprised of a spliced upstream with a nucleotide sequence of interest to be used as in gene therapy |
GB9720465D0 (en) | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
DK1895010T3 (da) | 1997-12-22 | 2011-11-21 | Oxford Biomedica Ltd | Vektorer baseret på virus for infektiøs hesteanæmi (eiav) |
GB2345062B (en) * | 1997-12-22 | 2001-07-25 | Oxford Biomedica Ltd | Retroviral vectors |
GB9803351D0 (en) | 1998-02-17 | 1998-04-15 | Oxford Biomedica Ltd | Anti-viral vectors |
AU763007B2 (en) | 1998-05-22 | 2003-07-10 | Oxford Biomedica (Uk) Limited | Retroviral delivery system |
US6555342B1 (en) * | 1998-06-03 | 2003-04-29 | Uab Research Foundation | Fusion protein delivery system and uses thereof |
US6958226B1 (en) * | 1998-09-11 | 2005-10-25 | The Children's Medical Center Corp. | Packaging cells comprising codon-optimized gagpol sequences and lacking lentiviral accessory proteins |
US20020002269A1 (en) | 1998-09-29 | 2002-01-03 | Jeffrey D. Milbrandt | Artemin, a neurotrophic factor |
US6506378B1 (en) * | 1998-12-16 | 2003-01-14 | Arch Development Corporation | Vesicular monoamine transporter gene therapy in Parkinson's disease |
-
2000
- 2000-10-06 GB GBGB0024550.6A patent/GB0024550D0/en not_active Ceased
-
2001
- 2001-10-05 EP EP01972317A patent/EP1337655B1/en not_active Expired - Lifetime
- 2001-10-05 JP JP2002532634A patent/JP4224295B2/ja not_active Expired - Lifetime
- 2001-10-05 DE DE60142576T patent/DE60142576D1/de not_active Expired - Lifetime
- 2001-10-05 DK DK01972317.0T patent/DK1337655T3/da active
- 2001-10-05 EP EP09014156A patent/EP2180057A1/en not_active Withdrawn
- 2001-10-05 PT PT01972317T patent/PT1337655E/pt unknown
- 2001-10-05 WO PCT/GB2001/004433 patent/WO2002029065A2/en active Application Filing
- 2001-10-05 AU AU2001292093A patent/AU2001292093A1/en not_active Abandoned
- 2001-10-05 CA CA002424738A patent/CA2424738A1/en not_active Abandoned
- 2001-10-05 AT AT01972317T patent/ATE474058T1/de active
- 2001-10-05 ES ES01972317T patent/ES2348277T3/es not_active Expired - Lifetime
-
2003
- 2003-04-07 US US10/408,456 patent/US7259015B2/en not_active Expired - Lifetime
-
2006
- 2006-07-14 US US11/486,914 patent/US20070025970A1/en not_active Abandoned
-
2008
- 2008-07-07 US US12/168,563 patent/US20090111106A1/en not_active Abandoned
-
2010
- 2010-10-06 CY CY20101100888T patent/CY1110817T1/el unknown
Also Published As
Publication number | Publication date |
---|---|
US20070025970A1 (en) | 2007-02-01 |
WO2002029065A2 (en) | 2002-04-11 |
EP2180057A1 (en) | 2010-04-28 |
US20090111106A1 (en) | 2009-04-30 |
JP4224295B2 (ja) | 2009-02-12 |
AU2001292093A1 (en) | 2002-04-15 |
US7259015B2 (en) | 2007-08-21 |
US20040013648A1 (en) | 2004-01-22 |
ATE474058T1 (de) | 2010-07-15 |
PT1337655E (pt) | 2010-08-25 |
DE60142576D1 (de) | 2010-08-26 |
CA2424738A1 (en) | 2002-04-11 |
ES2348277T3 (es) | 2010-12-02 |
GB0024550D0 (da) | 2000-11-22 |
EP1337655B1 (en) | 2010-07-14 |
JP2004520016A (ja) | 2004-07-08 |
CY1110817T1 (el) | 2015-06-10 |
WO2002029065A3 (en) | 2002-12-27 |
EP1337655A2 (en) | 2003-08-27 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DK1337655T3 (da) | Lentivirusvektorer til behandling af neurodegenerative sygdomme | |
DK1351703T3 (da) | Anvendelse af et polypeptid omfattende det ekstracellulære domæne af IL-20RA og IL-20RB til behandling af inflammation | |
BRPI0211200B8 (pt) | peptídeos efetivos no tratamento de tumores e outras condições que requerem a remoção ou destruição de células | |
DK1692158T3 (da) | VEGF-binding og understøttede peptider til behandling af hudsygdomme | |
DK1404324T4 (da) | Prolægemidler af GABA analoger, sammensætninger og anvendelser deraf | |
NO20035611L (no) | Sammensetning og fremgangsmate for kontroll ved behandling av hydrokarbon | |
DK1799703T3 (da) | Fremgangsmåde til fremstilling af blandinger af polypeptider ved hjælp af oprenset hydrogenbromid | |
DK1328269T5 (da) | Kombination af en CB1-receptorantagonist og sibutramin til behandling af fedme | |
BR9812371A (pt) | Tratamento de distúrbio de conduta | |
CY1108424T1 (el) | Μεθοδοι για τη βελτιωση της διαμορφωσης πρωτεϊνων με τη βοηθεια αναγωγικων μεσων | |
ATE327744T1 (de) | Neue verwendung von peptidverbindungen bei der behandlung von nicht-neuropathischem entzündungsschmerz | |
ATE428413T1 (de) | Neue verwendung von peptidverbindungen zur behandlung ovn zentralen neuropathischen schmerzen | |
BR9708114A (pt) | Análogos xantona para o tratamento de doenças infecciosas. | |
WO2003074073A3 (fr) | Genes impliques dans la regulation de l'angiogenese, preparations pharmaceutiques les contenant et leurs applications. | |
WO2002061076A1 (en) | Adiponectin-associated protein | |
DK0996435T3 (da) | Aminosyrederivater anvendelige til behandling af slagtilfælde | |
DK1778271T3 (da) | Peptid til behandling af herpesvirusinfektioner | |
ATE414156T1 (de) | Lipase-varianten | |
EP1549672A4 (en) | ABLATED SLAM DEPENDENT ENTRY | |
DK1425295T3 (da) | Modificerede peptider og deres anvendelse til behandling af autoimmune sygdomme | |
EP1760087A4 (en) | NEW PROTEIN COMPLEX AND USE OF THIS COMPLEX | |
NO20033401L (no) | Kjemiske forbindelser | |
DE10293089D2 (de) | Phosphinat-peptidanaloga als Inhibitoren der Procollagen-C-Proteinase (PCP) zur Behandlung von fibrotischen Erkrankungen | |
ATE397753T1 (de) | Screening-verfahren für eine prophylaktische und therapeutische substanz für eine nierenerkrankung | |
DK0971714T3 (da) | Metode til anvendelse af cyklooxygenase-2-inhibitorer til behandling og forebyggelse af demens |