JP2019524890A5 - - Google Patents

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JP2019524890A5
JP2019524890A5 JP2019530369A JP2019530369A JP2019524890A5 JP 2019524890 A5 JP2019524890 A5 JP 2019524890A5 JP 2019530369 A JP2019530369 A JP 2019530369A JP 2019530369 A JP2019530369 A JP 2019530369A JP 2019524890 A5 JP2019524890 A5 JP 2019524890A5
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nucleic acid
raav
promoter
item
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JP2019530369A
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JP7165410B2 (ja
JP2019524890A (ja
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Priority claimed from PCT/US2017/047589 external-priority patent/WO2018035451A1/en
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JP2019530369A 2016-08-19 2017-08-18 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物 Active JP7165410B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022111264A JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

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Application Number Priority Date Filing Date Title
US201662377297P 2016-08-19 2016-08-19
US62/377,297 2016-08-19
PCT/US2017/047589 WO2018035451A1 (en) 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus

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JP2022111264A Division JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

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JP2019524890A JP2019524890A (ja) 2019-09-05
JP2019524890A5 true JP2019524890A5 (enExample) 2020-12-24
JP7165410B2 JP7165410B2 (ja) 2022-11-04

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JP2019530369A Active JP7165410B2 (ja) 2016-08-19 2017-08-18 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
JP2022111264A Active JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

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US (2) US11207382B2 (enExample)
EP (1) EP3500278B1 (enExample)
JP (2) JP7165410B2 (enExample)
KR (2) KR102759317B1 (enExample)
CN (1) CN109843306A (enExample)
AU (1) AU2017313844B2 (enExample)
CA (1) CA3073137A1 (enExample)
ES (1) ES2982489T3 (enExample)
WO (1) WO2018035451A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
CA3046347A1 (en) * 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated Il-1ra cdnas
BR112020014625A2 (pt) 2018-01-17 2020-12-08 Meiragtx Uk Ii Limited Proteína de capsídeo de raav modificada para terapia gênica
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
JP2022520647A (ja) 2019-02-15 2022-03-31 サンガモ セラピューティクス, インコーポレイテッド 組み換えaav生産のための組成物および方法
RS66255B1 (sr) * 2019-07-15 2024-12-31 Meiragtx Uk Ii Ltd Modifikovani proteini aav kapsida za lečenje artritične bolesti
JP2023509443A (ja) * 2019-12-31 2023-03-08 スワンバイオ セラピューティクス リミテッド 改善されたaav-abcd1コンストラクトならびに副腎白質ジストロフィー(ald)および/または副腎脊髄ニューロパチー(amn)の処置または予防のための使用
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)

Family Cites Families (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156304A (en) 1990-12-20 2000-12-05 University Of Pittsburgh Of The Commonwealth System Of Higher Education Gene transfer for studying and treating a connective tissue of a mammalian host
US5747072A (en) 1993-07-30 1998-05-05 University Of Michigan Adenoviral-mediated gene transfer to synovial cells in vivo
JPH08508415A (ja) * 1993-12-14 1996-09-10 ユニバーシティー オブ ピッツバーグ オブ ザ コモンウェルス システム オブ ハイヤー エデュケーション 結合組織疾患の全身的遺伝子治療
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
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
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
ES2224375T3 (es) 1997-04-14 2005-03-01 Cell Genesys, Inc. Metodos para aumentar la eficacia del producto de aav recombinante.
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
PL343064A1 (en) 1998-03-20 2001-07-30 Benitec Australia Ltd Control of gene expression
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
AU783037B2 (en) 1999-05-28 2005-09-15 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (TNF) in the TNF-associated disorders
US6429001B1 (en) 2000-01-26 2002-08-06 Chiron Corporation Recombinant AAV packaging systems
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
DE10056210A1 (de) 2000-11-13 2002-05-29 Arimedes Biotechnology Gmbh Virales Expressionssystem
HUP0400698A3 (en) * 2001-02-06 2006-01-30 Merck Patent Gmbh Modified interleukin-1 receptor antagonist (il-1ra) with reduced immunogenicity
KR100472297B1 (ko) 2001-09-27 2005-03-07 주식회사 두산 리소인지질의 수용성 조성물
KR100471942B1 (ko) 2002-03-19 2005-03-09 주식회사 에스원 복합형 카드와 카드정보 등록 제어시스템
DE10318048A1 (de) 2003-04-17 2004-11-04 Axaron Bioscience Ag Nicht fluoreszierende, durch Proteolyse zur Fluoreszenz aktivierbare Reporterproteine und ihre Verwendung zur Detektion Protease-abhängiger Ereignisse
US8529885B2 (en) 2003-09-01 2013-09-10 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US7196112B2 (en) 2004-07-16 2007-03-27 Biogen Idec Ma Inc. Cell adhesion inhibitors
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
FR2891544A1 (fr) 2005-09-30 2007-04-06 Genethon Ass Loi De 1901 Substrat proteique pour la detection de l'activite calpaine 3
US20090105148A1 (en) 2006-03-23 2009-04-23 Caritas St. Elizabeth Medical Center Of Boston, Inc. Compositions and methods for treating myocardial infarction
EP2037943A4 (en) 2006-04-21 2010-03-31 Univ North Carolina TREATMENT OF BIND WEB DISEASES
US20080187576A1 (en) 2006-06-16 2008-08-07 University Of Florida Research Foundation, Inc. Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
US20080166762A1 (en) 2006-09-27 2008-07-10 Reliance Life Science Pvt.Ltd. HETEROLOGOUS PRODUCTION OF INTERLEUKIN 1 RECEPTOR ANTAGONIST (IL-1Ra) IN PICHIA PASTORIS
US7452696B2 (en) 2006-10-27 2008-11-18 National Taiwan University Recombinant plasmid and method for expressing hepatitis B viral antigens and virions in vivo
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
GB0708376D0 (en) * 2007-05-01 2007-06-06 Alligator Bioscience Ab Novel polypeptides and uses thereof
CA3001374A1 (en) * 2008-11-17 2010-05-20 Vgx Pharmaceuticals, Llc Antigens that elicit immune response against flavivirus and methods of using same
WO2010121010A2 (en) 2009-04-16 2010-10-21 President And Fellows Of Harvard College Methods for inhibiting starvation of a cell
EP2482814A4 (en) 2009-09-29 2013-04-03 Gilead Biologics Inc METHOD AND COMPOSITIONS FOR TREATING OKULAR FIBROSIS
DE202009017772U1 (de) 2009-12-10 2011-04-21 Orthogen Ag Kombinationspräparate mit Cytokin-Antagonist und Corticosteroid
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012047093A1 (en) 2010-10-05 2012-04-12 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of the sjögren's syndrome
KR101333958B1 (ko) 2010-10-20 2013-11-27 주식회사 한독 인간 인터루킨-1 수용체 길항제-하이브리드 Fc 융합단백질
US8736207B2 (en) 2011-01-03 2014-05-27 General Electric Company Method and system for power conversion
MX341578B (es) 2011-02-08 2016-08-25 Abbvie Inc Tratamiento de la osteoartritis y del dolor.
US20130021786A1 (en) 2011-07-21 2013-01-24 Noble Matthew D Utility bag lighting
ES2534782T3 (es) 2012-02-02 2015-04-28 Baylor College Of Medicine Sistema biológico de administración y expresión basado en adenovirus para uso en el tratamiento de la osteoartritis
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9878056B2 (en) * 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
CN103509100B (zh) 2012-06-15 2017-10-27 上海百迈博制药有限公司 一种白细胞介素‑1受体拮抗剂突变体
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
DK2948553T3 (da) 2013-01-25 2020-06-29 Baylor College Medicine Hjælper-afhængigt afgivelses- og ekspressionssystem til adenoviral genterapi
CA2909706C (en) 2013-04-17 2023-02-14 Genzyme Corporation Use of an il17 inhibitor for treating and preventing macular degeneration
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
EP4219727A3 (en) 2013-08-27 2023-09-06 Research Institute at Nationwide Children's Hospital Products and methods for treatment of amyotrophic lateral sclerosis
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
DE102013220859B4 (de) 2013-10-15 2016-09-08 Plasmidfactory Gmbh & Co. Kg Minicircles mit viralen Expressionskassetten und ihre Verwendung zur Transformation von Zellen zur Erzeugung rekombinanter Viren oder viraler Genvektoren
AU2015247777B2 (en) 2014-04-15 2019-03-21 Beacon Therapeutics Limited Codon optimized nucleic acid encoding a retinitis pigmentosa GTPase regulator (RPGR)
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
PT3137497T (pt) 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
US20200318080A1 (en) 2016-08-19 2020-10-08 Colorado State University Research Foundation Methods and compositions for treating equine conditions using recombinant self-complementary adeno-associated virus
US20210283222A1 (en) 2016-08-19 2021-09-16 Calimmune, Inc. Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus
CA3046347A1 (en) 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated Il-1ra cdnas

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