BR112018071167A2 - entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular - Google Patents

entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular

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Publication number
BR112018071167A2
BR112018071167A2 BR112018071167A BR112018071167A BR112018071167A2 BR 112018071167 A2 BR112018071167 A2 BR 112018071167A2 BR 112018071167 A BR112018071167 A BR 112018071167A BR 112018071167 A BR112018071167 A BR 112018071167A BR 112018071167 A2 BR112018071167 A2 BR 112018071167A2
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Brazil
Prior art keywords
muscular dystrophy
sarcoglycan
microrna
adeno
beta
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Application number
BR112018071167A
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English (en)
Inventor
Rodino-Klapac Louise
R. Mendell Jerry
Original Assignee
Research Institute At Nationwide Children's Hospital
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Publication date
Application filed by Research Institute At Nationwide Children's Hospital filed Critical Research Institute At Nationwide Children's Hospital
Publication of BR112018071167A2 publication Critical patent/BR112018071167A2/pt

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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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Abstract

trata-se de vetores de aav recombinantes que compreendem uma sequência de polinucleotídeos que compreende ß-sarcoglicano e métodos de uso dos vetores recombinantes para reduzir ou prevenir fibrose em um mamífero que sofre de uma distrofia muscular. também são descritas, no presente documento, terapias que compreendem administrar o vetor de aav (ou vetores de aav) que expressa ß-sarcoglicano e mir-29c a um mamífero que sofre de uma distrofia muscular.
BR112018071167A 2016-04-15 2017-04-14 entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular BR112018071167A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US201662433548P 2016-12-13 2016-12-13
PCT/US2017/027583 WO2017180976A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

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BR112018071167A2 true BR112018071167A2 (pt) 2019-02-05

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BR112018071167A BR112018071167A2 (pt) 2016-04-15 2017-04-14 entrega de beta-sarcoglicano e microrna-29 por vírus adenoassociado e tratamento de distrofia muscular

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US (3) US11358993B2 (pt)
EP (1) EP3442600B1 (pt)
JP (2) JP7079733B2 (pt)
KR (1) KR102502091B1 (pt)
CN (2) CN109121395B (pt)
AU (2) AU2017250756B2 (pt)
BR (1) BR112018071167A2 (pt)
CA (1) CA3021057A1 (pt)
CO (1) CO2018012082A2 (pt)
DK (1) DK3442600T3 (pt)
FI (1) FI3442600T3 (pt)
IL (1) IL262262B2 (pt)
LT (1) LT3442600T (pt)
MA (1) MA44681A (pt)
MX (2) MX2018012605A (pt)
MY (1) MY195439A (pt)
PT (1) PT3442600T (pt)
RS (1) RS65490B1 (pt)
SA (1) SA518400245B1 (pt)
SG (1) SG11201809029RA (pt)
WO (1) WO2017180976A1 (pt)
ZA (1) ZA201806863B (pt)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
EP3020403A1 (en) 2014-11-14 2016-05-18 Universitat de Valéncia Compounds for the treatment of myotonic dystrophy
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOMIC ENGINEERING WITH TYPE I CRISPRISMS IN EUKARYOTIC CELLS
MA45477A (fr) * 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
US11358993B2 (en) * 2016-04-15 2022-06-14 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
JP2021500029A (ja) * 2017-10-20 2021-01-07 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｎｔ−３遺伝子治療のための方法及び材料
CA3103485A1 (en) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
WO2020006458A1 (en) * 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP3598978B1 (en) 2018-07-26 2024-05-29 EXOFIX S.r.l. Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles
US20220119489A1 (en) * 2019-01-25 2022-04-21 Children's Medical Center Corporation Compositions and methods for treating muscular dystrophy and related disorders
US20230241252A1 (en) * 2019-02-26 2023-08-03 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
TW202120532A (zh) * 2019-08-21 2021-06-01 美國全美兒童醫院之研究學會 α肌聚糖之腺相關病毒載體遞送及肌肉萎縮症之治療
JP2023506030A (ja) * 2019-12-16 2023-02-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN118045206A (zh) * 2024-04-12 2024-05-17 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
AU678867B2 (en) 1993-11-09 1997-06-12 Medical College Of Ohio, The Stable cell lines capable of expressing the adeno-associated virus replication gene
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5672694A (en) * 1995-10-24 1997-09-30 University Of Iowa Research Foundation β-sarcoglycan nucleic acid sequence, and nucleic acid probes
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
CN1233291A (zh) 1996-09-06 1999-10-27 宾西法尼亚大学托管会 重组腺伴随病毒定向基因治疗的方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2557997T3 (es) 1997-09-05 2016-02-01 Genzyme Corporation Métodos para generar preparados de vectores AAV de título elevado sin virus auxiliar
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4520569B2 (ja) 2000-02-18 2010-08-04 照彦 豊岡 拡張型心筋症の遺伝子治療剤
AU5557501A (en) 2000-04-28 2001-11-12 Univ Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US20040126762A1 (en) * 2002-12-17 2004-07-01 Morris David W. Novel compositions and methods in cancer
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
US7883858B2 (en) 2005-01-27 2011-02-08 Institute For Systems Biology Methods for identifying and monitoring drug side effects
CA2625403A1 (en) 2005-10-17 2007-04-26 Institute For System Biology Tissue-and serum-derived glycoproteins and methods of their use
EP2520935A3 (en) 2006-08-09 2013-02-13 Homestead Clinical Corporation Organ-specific proteins and methods of their use
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
JP5600064B2 (ja) 2007-10-26 2014-10-01 アカデミシュ ジーケンハウス ライデン 筋障害を相殺するための手段と方法
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US8729041B2 (en) * 2008-12-03 2014-05-20 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
EP2833923A4 (en) 2012-04-02 2016-02-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
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
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6626829B2 (ja) 2014-01-21 2019-12-25 フレイエ ユニヴェルシテイト ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｔｅｉｔ Ｂｒｕｓｓｅｌ 筋特異的核酸調節エレメント並びにその方法及び使用
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
US11358993B2 (en) * 2016-04-15 2022-06-14 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
AU2021293197A1 (en) * 2020-06-15 2023-02-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies

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