JP7630176B2 - 操作されたカプシドを有するアデノ随伴ウイルス - Google Patents

操作されたカプシドを有するアデノ随伴ウイルス Download PDF

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JP7630176B2
JP7630176B2 JP2021558819A JP2021558819A JP7630176B2 JP 7630176 B2 JP7630176 B2 JP 7630176B2 JP 2021558819 A JP2021558819 A JP 2021558819A JP 2021558819 A JP2021558819 A JP 2021558819A JP 7630176 B2 JP7630176 B2 JP 7630176B2
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raav
aav5
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クリストファー エー． リード，
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テナヤ セラピューティクス， インコーポレイテッド
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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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    • 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
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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
    • C12N15/63—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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
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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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    • 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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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Crystallography & Structural Chemistry (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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JP2021558819A 2019-04-01 2020-03-31 操作されたカプシドを有するアデノ随伴ウイルス Active JP7630176B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962827576P 2019-04-01 2019-04-01
US62/827,576 2019-04-01
US202062984197P 2020-03-02 2020-03-02
US62/984,197 2020-03-02
PCT/US2020/026009 WO2020205889A1 (en) 2019-04-01 2020-03-31 Adeno-associated virus with engineered capsid

Publications (4)

Publication Number Publication Date
JP2022527199A JP2022527199A (ja) 2022-05-31
JP2022527199A5 JP2022527199A5 (https=) 2023-04-04
JPWO2020205889A5 JPWO2020205889A5 (https=) 2023-04-04
JP7630176B2 true JP7630176B2 (ja) 2025-02-17

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US (3) US20220154217A1 (https=)
EP (1) EP3947700A4 (https=)
JP (1) JP7630176B2 (https=)
KR (1) KR20210148232A (https=)
CN (1) CN114450411A (https=)
AU (1) AU2020253435A1 (https=)
CA (1) CA3135609A1 (https=)
IL (1) IL286831A (https=)
SG (1) SG11202110607WA (https=)
WO (1) WO2020205889A1 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
WO2021142267A1 (en) 2020-01-08 2021-07-15 Scarpmap Llc Methods and computing system for processing ultrasound image to determine health of subdermal tissue
IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
CN116096734A (zh) 2020-05-13 2023-05-09 沃雅戈治疗公司 Aav衣壳的向性的重定向
BR112023001336A2 (pt) * 2020-08-05 2023-02-14 Spacecraft Seven Llc Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina)
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
TW202237850A (zh) * 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
TW202246505A (zh) * 2021-03-05 2022-12-01 俄羅斯聯邦商亞那拜恩有限公司 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
US20250205363A1 (en) * 2021-12-01 2025-06-26 Shape Therapeutics Inc. Functional aav capsids for systemic administration
WO2023108129A1 (en) * 2021-12-10 2023-06-15 Spacecraft Seven, Llc Troponin c (tnnc1) gene therapy using aav vector
CA3254775A1 (en) * 2022-03-18 2023-09-21 University Of Florida Research Foundation, Incorporated METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
TW202400798A (zh) * 2022-04-11 2024-01-01 美商特納亞治療股份有限公司 斑菲素蛋白—２（ｐｌａｋｏｐｈｉｌｌｉｎ—２）基因療法之治療方法
WO2023230464A2 (en) * 2022-05-23 2023-11-30 New York University A murine model to test the effect of pkp2 mutations on living adult hearts and uses thereof
AR129733A1 (es) * 2022-06-28 2024-09-25 Voyager Therapeutics Inc Variantes de cápsides de aav y sus usos
WO2025024826A1 (en) * 2023-07-27 2025-01-30 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
WO2025147436A1 (en) * 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016513477A (ja) 2013-03-15 2016-05-16 ザ・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 二重グリカン結合性ａａｖベクターのための方法および組成物
WO2017201121A1 (en) 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs

Family Cites Families (142)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4962091A (en) 1986-05-23 1990-10-09 Syntex (U.S.A.) Inc. Controlled release of macromolecular polypeptides
US7070994B2 (en) 1988-03-21 2006-07-04 Oxford Biomedica (Uk) Ltd. Packaging cells
US5591624A (en) 1988-03-21 1997-01-07 Chiron Viagene, Inc. Retroviral packaging cell lines
CA1339354C (en) 1988-09-01 1997-08-26 The Whitehead Institute For Biomedical Research Recombinant retroviruses with amphotropic and ecotropic host ranges
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US5817491A (en) 1990-09-21 1998-10-06 The Regents Of The University Of California VSV G pseusdotyped retroviral vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
FR2688514A1 (fr) 1992-03-16 1993-09-17 Centre Nat Rech Scient Adenovirus recombinants defectifs exprimant des cytokines et medicaments antitumoraux les contenant.
DE4228457A1 (de) 1992-08-27 1994-04-28 Beiersdorf Ag Herstellung von heterodimerem PDGF-AB mit Hilfe eines bicistronischen Vektorsystems in Säugerzellen
CA2145093C (en) 1992-09-25 2007-04-10 Lawrence Leroy Kunz Therapeutic inhibitor of vascular smooth muscle cells
AU680459B2 (en) 1992-12-03 1997-07-31 Genzyme Corporation Gene therapy for cystic fibrosis
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
ES2249761T3 (es) 1993-06-24 2006-04-01 Advec Inc. Vectores de adenovirus para terapia genica.
EP1637608B1 (en) 1993-10-25 2009-07-22 CANJI, Inc. Recombinant adenoviral vector and methods of use
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
FR2722208B1 (fr) 1994-07-05 1996-10-04 Inst Nat Sante Rech Med Nouveau site interne d'entree des ribosomes, vecteur le contenant et utilisation therapeutique
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5928906A (en) 1996-05-09 1999-07-27 Sequenom, Inc. Process for direct sequencing during template amplification
FR2751345B1 (fr) 1996-07-16 1998-09-18 Univ Paris Curie Lignees d'encapsidation hautement productrices
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6306434B1 (en) 1997-02-12 2001-10-23 Chong Kun Dang Corp. Pharmaceutical composition comprising cyclosporin solid-state microemulsion
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES 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
CA2349838C (en) 1998-11-05 2011-06-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
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
JP3904451B2 (ja) 1999-06-01 2007-04-11 俊雄 北村 パッケージング細胞
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
ATE324321T1 (de) 2000-08-31 2006-05-15 Edwin Lundgren Steuervorrichtung für einen lenkdrachen an einem boot
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
WO2002098432A1 (en) 2001-06-01 2002-12-12 The Brigham And Women's Hospital, Inc. Methods of treating cardiac disorders
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
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
US7091029B2 (en) 2002-09-23 2006-08-15 Applied Genetics Technologies Corporation High titer recombinant AAV production
US7485291B2 (en) 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US7960100B1 (en) 2004-04-30 2011-06-14 Celera Corporation Colon cancer targets and uses thereof
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2007078599A2 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
US20070161031A1 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP2520935A3 (en) 2006-08-09 2013-02-13 Homestead Clinical Corporation Organ-specific proteins and methods of their use
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
WO2009126894A2 (en) 2008-04-11 2009-10-15 Massachusetts Eye And Ear Infirmary Methods and compositions for the diagnosis and treatment of angiogenic disorders
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
JP5988961B2 (ja) 2010-04-28 2016-09-07 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 心筋細胞を発生させるための方法
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス， インコーポレイテッド 新規のｄｎａ結合タンパク質及びその使用
HRP20171334T1 (hr) 2011-04-22 2017-11-17 The Regents Of The University Of California Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe
WO2013033213A1 (en) 2011-08-30 2013-03-07 The J. David Gladstone Institutes Methods for generating cardiomyocytes
US9441206B2 (en) 2011-10-28 2016-09-13 The University Of North Carolina At Chapel Hill Cell line for production of adeno-associated virus
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
ES2697249T3 (es) 2013-03-11 2019-01-22 Fond Telethon miR-204 y miR-211 y usos de los mismos
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
US10604771B2 (en) 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3039129B1 (en) 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3043822A1 (en) 2013-09-11 2016-07-20 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Compositions for preparing cardiomyocytes
EP3043866A4 (en) 2013-09-12 2017-05-17 President and Fellows of Harvard College Methods for inducing glucose uptake
WO2015042581A1 (en) 2013-09-23 2015-03-26 President And Fellows Of Harvard College Allele-specific rna silencing for the treatment of hypertrophic cardiomyopathy
US10144915B2 (en) 2013-10-23 2018-12-04 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Reprogramming fibroblasts into cardiomyocytes
EP3099333B8 (en) 2014-01-31 2020-12-30 Temple University of the Commonwealth System of Higher Education Bag3 as a target for therapy of heart failure
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US9287021B2 (en) * 2014-03-04 2016-03-15 Whirlpool Corporation Shelf brackets to conduct electricity to refrigerator shelves
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
DK3467111T3 (da) 2014-04-22 2022-03-14 Medizinische Hochschule Hannover LNCRNAS til terapi og diagnose af hjertehypertrofi
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated 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
JP6960396B2 (ja) 2015-04-07 2021-11-05 ザ ジェイ． デビッド グラッドストーン インスティテューツ、 ア テスタメンタリー トラスト エスタブリッシュド アンダー ザ ウィル オブ ジェイ． デビッド グラッドストーン 分裂終了細胞の細胞分裂を誘発するための方法
EP3286318A2 (en) 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
US20180296703A1 (en) 2015-08-17 2018-10-18 Temple University Of The Commonwealth System Of Higher Education Bag3 compositions and methods
CN108603176B (zh) 2015-11-01 2022-10-21 格里科贝克有限责任公司 无病毒细胞系及获得其的方法
WO2017083750A1 (en) 2015-11-11 2017-05-18 Intrexon Corporation Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
EP3436569A4 (en) 2016-03-30 2019-11-13 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone ADVANCED DIRECT CARDIAL PROGRAMMING
WO2017181162A1 (en) 2016-04-16 2017-10-19 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
WO2018005546A1 (en) 2016-06-27 2018-01-04 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Engineered cardiomyocytes and uses thereof
US11566243B2 (en) * 2016-07-18 2023-01-31 Jaan Biotherapeutics Llc Compositions and methods for treatment of cardiac diseases
CN109937258B (zh) 2016-10-21 2023-06-23 味之素株式会社 蛋白质的分泌产生方法
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018222503A1 (en) * 2017-05-31 2018-12-06 The Regents Of The University Of California Adeno-associated virus with variant capsid and methods of use thereof
CN107634856B (zh) 2017-09-18 2020-12-22 金华市智甄通信设备有限公司 一种路由器固件升级方法及系统
US12186369B2 (en) 2017-09-20 2025-01-07 The Regents Of The University Of California Gene therapy strategy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy
SG11202002276VA (en) 2017-09-20 2020-04-29 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
US12098385B2 (en) 2017-09-20 2024-09-24 The Regents Of The University Of California Gene therapy strategy to restore cardiac electrical and structural function in arrhythmogenic right ventricular cardiomyopathy
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
AU2019258830B2 (en) 2018-04-27 2025-12-18 Universität Heidelberg Modified AAV capsid polypeptides for treatment of muscular diseases
CA3111076A1 (en) 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1
US20220088052A1 (en) 2019-01-22 2022-03-24 Koninklijke Nederlandse Akademie Van Wetenschappen Tfap2 inhibition for treating cardiac disease involving fibro-fatty replacement
CA3134429A1 (en) 2019-03-28 2020-10-01 Esteve Pharmaceuticals, S.A. Methods for the manufacture of recombinant viral vectors
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP3959226A2 (en) 2019-04-26 2022-03-02 Sangamo Therapeutics, Inc. Engineering aav
WO2020223279A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. VECTORIZED ANTIBODIES (vAb) AND USES THEREOF
WO2020236982A1 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Aav delivery of nucleobase editors
AU2020299026A1 (en) 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
RU2751592C2 (ru) 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
WO2021041473A1 (en) 2019-08-27 2021-03-04 The Trustees Of Columbia University In The City Of New York Engineered exosomes for targeted delivery
KR20220066914A (ko) 2019-09-20 2022-05-24 유씨엘 비즈니스 리미티드 부정맥 유발성 우심실 심근병증의 유전자 요법 조성물 및 치료
US20220403414A1 (en) 2019-10-16 2022-12-22 Wuxi Apptec (Shanghai) Co., Ltd. Novel aav variant
MX2022004771A (es) 2019-10-22 2022-10-07 Applied Genetic Tech Corporation Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72.
CA3159964A1 (en) 2019-12-04 2021-06-10 Ac Immune Sa Novel molecules for therapy and diagnosis
AU2020408225A1 (en) 2019-12-20 2022-07-14 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
WO2021183825A1 (en) 2020-03-12 2021-09-16 California Institute Of Technology Adeno-associated viral capsids with expanded sizes
WO2021187380A1 (ja) 2020-03-16 2021-09-23 国立大学法人大阪大学 不整脈源性心筋症患者由来の多能性幹細胞およびその利用ならびに不整脈源性心筋症治療用医薬
CN116406297A (zh) 2020-08-05 2023-07-07 阿斯克肋匹奥生物制药公司 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法
JP2023538519A (ja) 2020-08-07 2023-09-08 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを使用したプラコフィリン－２（ｐｋｐ２）遺伝子治療
EP3957650A1 (en) 2020-08-17 2022-02-23 Themis Bioscience GmbH Co-stimulatory 4-1bbl ectodomain polypeptides for immunomodulation
WO2022066898A2 (en) 2020-09-23 2022-03-31 Codiak Biosciences, Inc. Methods of producing extracellular vesicles
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
IL301255A (en) 2020-10-09 2023-05-01 UCB Biopharma SRL Nucleic acid structures, viral vectors and viral particles
WO2022079082A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for simultaneous gene activation
WO2022098765A1 (en) 2020-11-03 2022-05-12 The Board Of Trustees Of The University Of Illinois Split prime editing platforms
KR20230127221A9 (ko) 2020-12-01 2024-12-06 로카나바이오 인크. Cag 반복 질환을 치료하기 위한 rna 표적화 조성물 및 방법
US20240108751A1 (en) 2020-12-01 2024-04-04 Locanabio, Inc. Rna-targeting compositions and methods for treating myotonic dystrophy type 1
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
WO2022173847A2 (en) 2021-02-09 2022-08-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
KR20230159471A (ko) 2021-03-19 2023-11-21 유씨엘 비즈니스 리미티드 우심실 부정맥성 심근병증의 유전자 치료 조성물 및 치료
AU2022242086A1 (en) 2021-03-26 2023-10-19 Specific Biologics Inc. Targeting oncogentic mutations with dual-cleaving endonuclease
EP4320144A4 (en) 2021-04-05 2025-04-09 Janssen Biotech, Inc. T CELL RECEPTORS CALR AND JAK2
US20240173355A1 (en) 2021-04-05 2024-05-30 The Board Of Trustees Of The Leland Stanford Junior University Gene correction for rag2 deficiency in human stem cells
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2023200742A2 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Capsids for plakophillin-2 gene therapy
TW202400798A (zh) 2022-04-11 2024-01-01 美商特納亞治療股份有限公司 斑菲素蛋白—２（ｐｌａｋｏｐｈｉｌｌｉｎ—２）基因療法之治療方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016513477A (ja) 2013-03-15 2016-05-16 ザ・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 二重グリカン結合性ａａｖベクターのための方法および組成物
WO2017201121A1 (en) 2016-05-17 2017-11-23 University Of Florida Research Foundation, Inc. Recombinant aav for gene therapy in lungs

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