ES2683695T3 - Repeticiones terminales invertidas restrictivas para vectores virales - Google Patents
Repeticiones terminales invertidas restrictivas para vectores virales Download PDFInfo
- Publication number
- ES2683695T3 ES2683695T3 ES11733293T ES11733293T ES2683695T3 ES 2683695 T3 ES2683695 T3 ES 2683695T3 ES 11733293 T ES11733293 T ES 11733293T ES 11733293 T ES11733293 T ES 11733293T ES 2683695 T3 ES2683695 T3 ES 2683695T3
- Authority
- ES
- Spain
- Prior art keywords
- inverse
- restrictive
- viral vectors
- terminal repeats
- itr
- 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.)
- Active
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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14161—Methods of inactivation or attenuation
- C12N2750/14162—Methods of inactivation or attenuation by genetic engineering
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14311—Parvovirus, e.g. minute virus of mice
- C12N2750/14351—Methods of production or purification of viral material
- C12N2750/14352—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2820/00—Vectors comprising a special origin of replication system
- C12N2820/60—Vectors comprising a special origin of replication system from viruses
Abstract
Un polinucleótido que comprende al menos una repetición terminal invertida (ITR) del virus adenoasociado (AAV), en el que dicha ITR comprende la secuencia de nucleótidos:**Fórmula**
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US29418110P | 2010-01-12 | 2010-01-12 | |
PCT/US2011/020939 WO2011088081A1 (en) | 2010-01-12 | 2011-01-12 | Restrictive inverted terminal repeats for viral vectors |
Publications (1)
Publication Number | Publication Date |
---|---|
ES2683695T3 true ES2683695T3 (es) | 2018-09-27 |
Family
ID=44304614
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ES11733293T Active ES2683695T3 (es) | 2010-01-12 | 2011-01-12 | Repeticiones terminales invertidas restrictivas para vectores virales |
Country Status (4)
Country | Link |
---|---|
US (5) | US9169494B2 (es) |
EP (1) | EP2524037B1 (es) |
ES (1) | ES2683695T3 (es) |
WO (1) | WO2011088081A1 (es) |
Families Citing this family (91)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
EP2699680B1 (en) | 2011-04-21 | 2017-12-27 | University of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
TWI702955B (zh) | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
EP2970966A4 (en) * | 2013-03-15 | 2016-10-26 | Univ North Carolina | SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES |
EP3633041A3 (en) | 2013-09-26 | 2020-07-29 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
US10000741B2 (en) | 2014-03-17 | 2018-06-19 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
ES2856403T3 (es) | 2014-03-18 | 2021-09-27 | Univ Massachusetts | Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica |
WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
EP3200830B1 (en) | 2014-10-03 | 2020-09-09 | University of Massachusetts | High efficiency library-identified aav vectors |
US10480011B2 (en) * | 2014-10-21 | 2019-11-19 | University Of Massachusetts | Recombinant AAV variants and uses thereof |
CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
US10570395B2 (en) | 2014-11-14 | 2020-02-25 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
BR112017018846A2 (pt) | 2015-03-02 | 2018-07-31 | Adverum Biotechnologies, Inc. | composições e métodos para entrega intravítrea de polinucleotídeos a cones retinianos. |
CA3021949C (en) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
CA2983593A1 (en) | 2015-05-14 | 2016-11-17 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
WO2016210097A1 (en) * | 2015-06-23 | 2016-12-29 | The Trustees Of Columbia University In The City Of New York | Novel equine parvovirus and uses thereof |
WO2017066579A1 (en) | 2015-10-14 | 2017-04-20 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
US20200181644A1 (en) * | 2015-10-22 | 2020-06-11 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial aav3 capsid library |
US11253576B2 (en) | 2015-10-22 | 2022-02-22 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
US11426469B2 (en) | 2015-10-22 | 2022-08-30 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
EP3411059A4 (en) | 2016-02-02 | 2019-10-16 | University Of Massachusetts | METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM |
EP3413928B1 (en) | 2016-02-12 | 2022-04-20 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHODS FOR SELECTIVE INHIBITION OF EXPRESSION OF GRAINHEAD PROTEIN |
WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
KR102427379B1 (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
EP3500696A4 (en) | 2016-08-16 | 2020-04-08 | Bluebird Bio, Inc. | IL-10 RECEPTOR ALPHA HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS FOR USE |
EP3504335A4 (en) | 2016-08-23 | 2020-04-15 | Bluebird Bio, Inc. | TIM3 HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
CA3035522A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
KR102451510B1 (ko) | 2016-09-08 | 2022-10-07 | 2세븐티 바이오, 인코포레이티드 | Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법 |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
EP3526333A4 (en) | 2016-10-13 | 2020-07-29 | University of Massachusetts | AAV CAPSIDE DESIGNS |
MA46543A (fr) | 2016-10-17 | 2019-08-21 | Bluebird Bio Inc | Variants de l'endonucléase tgfbéta r2, compositions et procédés d'utilisation |
DK3541833T3 (da) | 2016-11-17 | 2024-04-02 | 2Seventy Bio Inc | TGFß signalkonverter |
EP3583203B1 (en) | 2017-02-15 | 2023-11-01 | 2seventy bio, Inc. | Donor repair templates multiplex genome editing |
JP7237843B2 (ja) | 2017-02-21 | 2023-03-13 | ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. | 修飾されたaavキャプシドタンパク質およびその使用 |
JP2020518258A (ja) | 2017-05-05 | 2020-06-25 | 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. | 筋萎縮性側索硬化症(als)治療組成物および方法 |
CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
AU2018264996A1 (en) | 2017-05-09 | 2019-12-05 | University Of Massachusetts | Methods of treating Amyotrophic Lateral Sclerosis (ALS) |
CA3064014A1 (en) | 2017-05-25 | 2018-11-29 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | NETWORK EQUIPMENT TRACK GUIDE SYSTEM |
JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
EP3665289A1 (en) * | 2017-08-09 | 2020-06-17 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof |
CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
WO2019070974A1 (en) | 2017-10-04 | 2019-04-11 | Bluebird Bio, Inc. | PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
EP3697905A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
US11905524B2 (en) * | 2018-03-06 | 2024-02-20 | University Of Florida Research Foundation, Incorporated | AAV chimeras |
JP2021527409A (ja) | 2018-06-14 | 2021-10-14 | ブルーバード バイオ, インコーポレイテッド | Cd79aキメラ抗原受容体 |
CN112639110A (zh) * | 2018-06-22 | 2021-04-09 | 阿斯克肋匹奥生物制药公司 | 用于基因递送以在细胞内持续存在的载体 |
CA3108799A1 (en) * | 2018-08-09 | 2020-02-13 | Bioverativ Therapeutics Inc. | Nucleic acid molecules and uses thereof for non-viral gene therapy |
KR20210102870A (ko) | 2018-08-30 | 2021-08-20 | 테나야 테라퓨틱스, 인코포레이티드 | 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍 |
WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
WO2020092904A1 (en) * | 2018-11-02 | 2020-05-07 | Nikegen, Llc | Recombinant parvoviral vectors and method of making and use thereof |
WO2021007515A1 (en) | 2019-07-11 | 2021-01-14 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with micrornas and other factors |
CA3166987A1 (en) * | 2020-01-07 | 2021-07-15 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats and methods of their use as promoters |
TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
KR20220167324A (ko) | 2020-04-10 | 2022-12-20 | 솔라 바이오사이언시즈 엘엘씨 | 단백질 응집 장애의 치료를 위한 조성물 및 방법 |
JP2023524414A (ja) | 2020-04-28 | 2023-06-12 | ソラ・バイオサイエンシズ・エルエルシー | Tdp-43プロテイノパチーの処置のための組成物および方法 |
EP4153760A1 (en) * | 2020-05-21 | 2023-03-29 | Nysnobio GT, LLC | Gene therapy delivery of parkin mutants having increased activity to treat parkinson's disease |
CN116096737A (zh) | 2020-06-05 | 2023-05-09 | 索拉生物科学有限公司 | 用于治疗突触核蛋白病的组合物和方法 |
JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Dna分子の組成物、その作製方法、及びその使用方法 |
US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
CA3199433A1 (en) | 2020-10-26 | 2022-05-05 | Sola Biosciences Llc | Compositions and methods for the treatment of alzheimer's disease |
IL309957A (en) | 2021-07-14 | 2024-03-01 | 2Seventy Bio Inc | Transgenic T cell receptors attached to antibody binding sites |
WO2023060248A1 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of p53-mediated cancers |
WO2023060221A2 (en) | 2021-10-08 | 2023-04-13 | Sola Biosciences Llc | Compositions and methods for the treatment of proteopathies |
WO2023196997A2 (en) | 2022-04-08 | 2023-10-12 | 2Seventy Bio, Inc. | Multipartite receptor and signaling complexes |
WO2023230512A1 (en) | 2022-05-26 | 2023-11-30 | 2Seventy Bio, Inc. | Compositions for maintaining lentiviral vector and uses thereof |
CN117802161A (zh) * | 2022-06-30 | 2024-04-02 | 苏州吉恒基因科技有限公司 | 精准重组腺相关病毒载体及其用途 |
Family Cites Families (33)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
WO1988002026A1 (en) | 1986-09-08 | 1988-03-24 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
WO1990005142A1 (en) | 1988-11-10 | 1990-05-17 | Imperial Cancer Research Technology Ltd. | Polypeptides |
US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
WO1996040272A1 (en) | 1995-06-07 | 1996-12-19 | University Of North Carolina At Chapel Hill | Aav transduction of myoblasts |
ES2279531T3 (es) | 1995-12-15 | 2007-08-16 | Intronn, Inc. | Moleculas terapeuticas generadas por corte y empalme en trans. |
US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
AU762220B2 (en) | 1998-05-28 | 2003-06-19 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | AAV5 vector and uses thereof |
US6984517B1 (en) | 1998-05-28 | 2006-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector and uses thereof |
NZ511171A (en) | 1998-09-22 | 2004-02-27 | Univ Florida | Methods for large-scale production of recombinant AAV vectors |
DK1127150T3 (da) | 1998-11-05 | 2007-09-24 | Univ Pennsylvania | Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse |
ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
DE19905501B4 (de) | 1999-02-10 | 2005-05-19 | MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie | Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels |
DE60117550T2 (de) | 2000-06-01 | 2006-12-07 | University Of North Carolina At Chapel Hill | Doppelsträngige parvovirus-vektoren |
AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
US20030129203A1 (en) * | 2001-08-27 | 2003-07-10 | Nautilus Biotech S.A. | Mutant recombinant adeno-associated viruses |
WO2003042361A2 (en) * | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
AU2003223766A1 (en) | 2002-04-30 | 2003-11-17 | University Of North Carolina At Chapel Hill | Secretion signal vectors |
ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
-
2011
- 2011-01-12 WO PCT/US2011/020939 patent/WO2011088081A1/en active Application Filing
- 2011-01-12 US US13/521,448 patent/US9169494B2/en active Active
- 2011-01-12 ES ES11733293T patent/ES2683695T3/es active Active
- 2011-01-12 EP EP11733293.2A patent/EP2524037B1/en active Active
-
2015
- 2015-10-26 US US14/922,935 patent/US10233428B2/en active Active
-
2019
- 2019-02-08 US US16/271,163 patent/US10858632B2/en active Active
-
2020
- 2020-11-19 US US16/953,109 patent/US11542478B2/en active Active
-
2022
- 2022-12-01 US US18/060,799 patent/US11939597B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
US10858632B2 (en) | 2020-12-08 |
US20160102297A1 (en) | 2016-04-14 |
US9169494B2 (en) | 2015-10-27 |
US20130109742A1 (en) | 2013-05-02 |
US11939597B2 (en) | 2024-03-26 |
US11542478B2 (en) | 2023-01-03 |
EP2524037B1 (en) | 2018-05-16 |
US20190185823A1 (en) | 2019-06-20 |
EP2524037A4 (en) | 2014-01-15 |
US20210071148A1 (en) | 2021-03-11 |
EP2524037A1 (en) | 2012-11-21 |
US10233428B2 (en) | 2019-03-19 |
WO2011088081A1 (en) | 2011-07-21 |
US20230167418A1 (en) | 2023-06-01 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
ES2683695T3 (es) | Repeticiones terminales invertidas restrictivas para vectores virales | |
TR201901582T4 (tr) | Kapsid | |
DK2721819T3 (da) | Entropikodning, som understøtter modusomskiftning | |
EA201492223A1 (ru) | Новые диазаспироциклоалканы и азаспироциклоалканы | |
BR112018011881A2 (pt) | proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus | |
CL2016001580A1 (es) | Uso de compuestos derivados de indol para tratar una infección por flavivirus; compuestos derivados de indol; composiciones farmacéuticas; y uso de una combinación. | |
CL2014002198A1 (es) | Composiciones de vector de virus adenoasociado (aav) y métodos para transferencia de genes a células, órganos y tejidos | |
ES2566033T3 (es) | Parvovirus recombinante atenuado | |
PE20150163A1 (es) | Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav | |
PE20151433A1 (es) | Nucleosidos sustituidos, nucleotidos y analogos de los mismos | |
EA201590292A1 (ru) | Соединения, которые являются sip-модулирующими агентами и/или atx-модулирующими агентами | |
CL2014001641A1 (es) | Compuestos derivados de nucleosidos sustituidos, nucleotidos y analogos de los mismos; composicion farmaceutica; y uso para mejorar o tratar una infeccion viral. | |
CL2014002392A1 (es) | Uso de nucleosidos, nucleotidos sustituidos y análogos de los mismos para el tratamiento de una infeccion viral. | |
DK2900686T3 (da) | Aav-vektorer targeteret til oligodendrocytter | |
JP2014526140A5 (es) | ||
JP2016525888A5 (es) | ||
UA107791C2 (en) | Pesticidal compositions | |
CL2012002969A1 (es) | Vector viral adenoasociado recombinante (raav) que comprende promotor de rodopsina quinasa humana especifica del fotorreceptor, o promotor de beta-actina de pollo cmv quimerico truncado ubicuo unido a al menos un adn que codifica una guanilato ciclasa; virion o particula viral infecciosa que comprende el vector; celula huesped; composicion que comprende el vector. | |
UA111626C2 (uk) | Похідні дигідрохінолін-2-ону | |
BRPI1007447A2 (pt) | catalisador para óxido de etileno com teor otimizado de césio. | |
DK2361324T3 (da) | Elektrode, der er egnet som hydrogenudviklende katode | |
BR112019009033A2 (pt) | métodos de purificação de vírus adenoassociado | |
BR112013029966A2 (pt) | oligodesoxinucleotídeo imunoestimulatório não metilado, vetor, e, vacina para prevenir ou combater uma doença infecciosa | |
BR112015011224A2 (pt) | misturas de reação de pcr e métodos de usar as mesmas | |
FR2968366B1 (fr) | Clip de fixation elastique. |