JP7237843B2 - 修飾されたaavキャプシドタンパク質およびその使用 - Google Patents
修飾されたaavキャプシドタンパク質およびその使用 Download PDFInfo
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- JP7237843B2 JP7237843B2 JP2019546041A JP2019546041A JP7237843B2 JP 7237843 B2 JP7237843 B2 JP 7237843B2 JP 2019546041 A JP2019546041 A JP 2019546041A JP 2019546041 A JP2019546041 A JP 2019546041A JP 7237843 B2 JP7237843 B2 JP 7237843B2
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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- C12N15/09—Recombinant DNA-technology
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/1034—Isolating an individual clone by screening libraries
- C12N15/1037—Screening libraries presented on the surface of microorganisms, e.g. phage display, E. coli display
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- 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
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N2750/14145—Special targeting system for viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Ophthalmology & Optometry (AREA)
- General Chemical & Material Sciences (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762461770P | 2017-02-21 | 2017-02-21 | |
| US62/461,770 | 2017-02-21 | ||
| US201862625486P | 2018-02-02 | 2018-02-02 | |
| US62/625,486 | 2018-02-02 | ||
| PCT/US2018/019050 WO2018156654A1 (en) | 2017-02-21 | 2018-02-21 | Modified aav capsid proteins and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020508667A JP2020508667A (ja) | 2020-03-26 |
| JP2020508667A5 JP2020508667A5 (OSRAM) | 2021-04-08 |
| JP7237843B2 true JP7237843B2 (ja) | 2023-03-13 |
Family
ID=63253351
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546041A Active JP7237843B2 (ja) | 2017-02-21 | 2018-02-21 | 修飾されたaavキャプシドタンパク質およびその使用 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US10793606B2 (OSRAM) |
| EP (1) | EP3585883A4 (OSRAM) |
| JP (1) | JP7237843B2 (OSRAM) |
| AU (1) | AU2018224044B2 (OSRAM) |
| CA (2) | CA3114549A1 (OSRAM) |
| WO (1) | WO2018156654A1 (OSRAM) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| EP3633041A3 (en) | 2013-09-26 | 2020-07-29 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| CA3075656A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| US11723988B2 (en) | 2017-10-20 | 2023-08-15 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector |
| WO2019158619A1 (en) * | 2018-02-15 | 2019-08-22 | Bjoerklund Tomas | Modified viral capsids |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020072451A1 (en) | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| EP3867359A4 (en) * | 2018-10-17 | 2022-09-21 | Children's Medical Research Institute | NUCLEIC ACID MOLECULES AND AAV VECTOR SELECTION METHODS |
| US20220133909A1 (en) | 2019-01-23 | 2022-05-05 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| US11141425B2 (en) | 2019-06-27 | 2021-10-12 | University Of Florida Research Foundation, Incorporated | Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid |
| JP2022550435A (ja) | 2019-10-04 | 2022-12-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えaavの改善された治療的使用のための方法 |
| IL294781A (en) | 2020-02-14 | 2022-09-01 | Ultragenyx Pharmaceutical Inc | Gene therapy for treating cdkl5 deficiency disorder |
| EP4121544A1 (en) | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| JP2023520402A (ja) | 2020-03-31 | 2023-05-17 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | プロピオン酸血症を処置するための遺伝子治療 |
| US20230233708A1 (en) * | 2020-04-14 | 2023-07-27 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated delivery and transduction with polyvinyl alcohol |
| CN111729078B (zh) * | 2020-07-24 | 2020-11-20 | 苏州世诺生物技术有限公司 | 鸡传染性贫血病毒基因工程疫苗 |
| CA3190214A1 (en) | 2020-07-29 | 2022-02-03 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| KR20230107285A (ko) * | 2020-11-10 | 2023-07-14 | 에이버맥스 바이오파마 인크. | 조작된 바이러스 캡시드 및 사용 방법 |
| TW202235618A (zh) * | 2021-01-05 | 2022-09-16 | 美國馬友醫藥教育研究基金會 | 治療眼內壓相關疾患 |
| WO2023019168A1 (en) | 2021-08-11 | 2023-02-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| AU2022358779A1 (en) | 2021-10-08 | 2024-04-18 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| TW202342740A (zh) | 2022-03-07 | 2023-11-01 | 美商奧崔基尼克斯製藥公司 | 改良的批量aav生產系統和方法 |
| AU2024234833A1 (en) | 2023-03-10 | 2025-10-23 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2024229049A1 (en) | 2023-05-01 | 2024-11-07 | University Of Florida Research Foundation, Incorporated | Dual aav vectors for treating stargardt disease |
| WO2025049993A1 (en) | 2023-08-31 | 2025-03-06 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025171227A1 (en) | 2024-02-08 | 2025-08-14 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025235555A1 (en) * | 2024-05-06 | 2025-11-13 | University Of Florida Research Foundation, Incorporated | Rational design of aav vectors with limited liver targeting |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010011404A2 (en) | 2008-05-20 | 2010-01-28 | Eos Neuroscience, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| WO2015121501A1 (en) | 2014-02-17 | 2015-08-20 | King's College London | Adeno-associated virus vector |
| WO2015134643A1 (en) | 2014-03-04 | 2015-09-11 | University Of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| JP2016520311A (ja) | 2013-05-21 | 2016-07-14 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
| JP2016533709A (ja) | 2013-09-26 | 2016-11-04 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5962313A (en) | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
| US6156303A (en) * | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
| CA2444483A1 (en) | 2001-04-26 | 2002-11-07 | Board Of Regents, The University Of Texas System | Diagnostic imaging compositions, their methods of synthesis and use |
| AU2002362646A1 (en) | 2001-10-09 | 2003-04-22 | The Johns Hopkins University | A phosphatase associated with metastasis |
| US20050106700A1 (en) | 2001-10-11 | 2005-05-19 | Tsuyoshi Nomura | Method of purifying recombinant fused protein and method of producing protein using the same |
| US20030225260A1 (en) | 2002-04-30 | 2003-12-04 | Snyder Richard O. | Production of recombinant AAV virions |
| US20060093589A1 (en) | 2004-02-19 | 2006-05-04 | Warrington Kenneth H | Vp2-modified raav vector compositions and uses therefor |
| WO2003093479A1 (en) | 2002-05-01 | 2003-11-13 | University Of Florida Research Foundation, Inc. | Improved raav expression systems and methods for enhancing transduction of mammalian neural cells |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| AU2003265855A1 (en) | 2002-08-28 | 2004-03-19 | University Of Florida | Modified aav |
| US20040219575A1 (en) | 2002-12-26 | 2004-11-04 | Toomas Neuman | Methods and compositions for the diagnosis, prognosis, and treatment of cancer |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| DK2357189T3 (en) * | 2003-06-19 | 2017-06-19 | Genzyme Corp | AAV virions with reduced immunoreactivity and applications thereof |
| ES2874298T3 (es) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| ES2428218T3 (es) | 2005-04-07 | 2013-11-06 | The Trustees Of The University Of Pennsylvania | Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas |
| WO2007089632A2 (en) | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| KR20090114430A (ko) | 2007-02-02 | 2009-11-03 | 베일러 리서치 인스티튜트 | 항원-제시 세포 상에서 발현된 dcir에 대한 항원 표적화에 기초한 백신 |
| TWI422594B (zh) | 2007-02-02 | 2014-01-11 | Baylor Res Inst | 經由樹狀細胞去唾液酸糖蛋白受體(dc-asgpr)接合抗原呈現細胞之藥劑 |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| EP2191001B1 (en) | 2007-04-09 | 2016-06-08 | University of Florida Research Foundation, Inc. | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| EP2396343B1 (en) * | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| US9169494B2 (en) | 2010-01-12 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| EP2634253B1 (en) | 2010-10-27 | 2016-05-11 | Jichi Medical University | Adeno-associated virus virions for transferring genes into neural cells |
| WO2012064960A2 (en) | 2010-11-10 | 2012-05-18 | Fred Hutchinson Cancer Research Center | Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination |
| DK2673289T3 (da) * | 2011-02-10 | 2023-07-24 | Univ North Carolina Chapel Hill | Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf |
| AU2013216382B2 (en) | 2012-01-31 | 2018-08-02 | Hadasit Medical Research Services And Development Ltd. | Methods of selecting retinal pigmented epithelial cells |
| ES2862912T3 (es) * | 2012-04-18 | 2021-10-08 | Childrens Hospital Philadelphia | Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CN110885854A (zh) | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| EP3066195A4 (en) | 2013-11-08 | 2017-06-28 | The Board of Trustees of the University of Arkansas | Adeno-associated virus "x" oncogene |
| WO2015126972A1 (en) | 2014-02-21 | 2015-08-27 | University Of Florida Research Foundation, Inc. | Methods and compositions for gene delivery to on bipolar cells |
| US11000597B2 (en) | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | Engineered receptor/ligand system for delivery of therapeutic agents |
| WO2016133917A1 (en) | 2015-02-16 | 2016-08-25 | University Of Florida Research Foundation | Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | 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The Regents Of The University Of California | アデノ随伴ウイルス変異体及びその使用方法 |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| US11266748B2 (en) | 2015-07-02 | 2022-03-08 | University Of Florida Research Foundation, Incorporated | Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| US11124546B2 (en) * | 2015-12-14 | 2021-09-21 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| EP4010357A2 (en) | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
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Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010011404A2 (en) | 2008-05-20 | 2010-01-28 | Eos Neuroscience, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| JP2016520311A (ja) | 2013-05-21 | 2016-07-14 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | キャプシド修飾rAAV3ベクター組成物およびヒト肝がんの遺伝子治療における使用方法 |
| JP2016533709A (ja) | 2013-09-26 | 2016-11-04 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー |
| WO2015121501A1 (en) | 2014-02-17 | 2015-08-20 | King's College London | Adeno-associated virus vector |
| WO2015134643A1 (en) | 2014-03-04 | 2015-09-11 | University Of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
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| CA3054131A1 (en) | 2018-08-30 |
| US20210061863A1 (en) | 2021-03-04 |
| US11767346B2 (en) | 2023-09-26 |
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| EP3585883A1 (en) | 2020-01-01 |
| US11332502B2 (en) | 2022-05-17 |
| AU2018224044B2 (en) | 2024-01-25 |
| US20220267383A1 (en) | 2022-08-25 |
| US20200002386A1 (en) | 2020-01-02 |
| US10793606B2 (en) | 2020-10-06 |
| AU2018224044A1 (en) | 2019-09-19 |
| WO2018156654A1 (en) | 2018-08-30 |
| CA3114549A1 (en) | 2018-08-30 |
| JP2020508667A (ja) | 2020-03-26 |
| CA3054131C (en) | 2021-05-25 |
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