JP2019528793A5 - - Google Patents

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Publication number
JP2019528793A5
JP2019528793A5 JP2019538714A JP2019538714A JP2019528793A5 JP 2019528793 A5 JP2019528793 A5 JP 2019528793A5 JP 2019538714 A JP2019538714 A JP 2019538714A JP 2019538714 A JP2019538714 A JP 2019538714A JP 2019528793 A5 JP2019528793 A5 JP 2019528793A5
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JP
Japan
Prior art keywords
vector
polynucleotide
adeno
promoter
associated virus
Prior art date
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JP2019538714A
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English (en)
Japanese (ja)
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JP2019528793A (ja
JP7059285B2 (ja
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Priority claimed from PCT/EP2017/074081 external-priority patent/WO2018060097A1/en
Publication of JP2019528793A publication Critical patent/JP2019528793A/ja
Publication of JP2019528793A5 publication Critical patent/JP2019528793A5/ja
Application granted granted Critical
Publication of JP7059285B2 publication Critical patent/JP7059285B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2019538714A 2016-09-30 2017-09-22 ムコ多糖症の治療のためのアデノ随伴ウイルスベクター Expired - Fee Related JP7059285B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16382450 2016-09-30
EP16382450.1 2016-09-30
PCT/EP2017/074081 WO2018060097A1 (en) 2016-09-30 2017-09-22 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

Publications (3)

Publication Number Publication Date
JP2019528793A JP2019528793A (ja) 2019-10-17
JP2019528793A5 true JP2019528793A5 (enExample) 2020-11-12
JP7059285B2 JP7059285B2 (ja) 2022-04-25

Family

ID=57123944

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019538714A Expired - Fee Related JP7059285B2 (ja) 2016-09-30 2017-09-22 ムコ多糖症の治療のためのアデノ随伴ウイルスベクター

Country Status (10)

Country Link
US (1) US11338046B2 (enExample)
EP (1) EP3519569B1 (enExample)
JP (1) JP7059285B2 (enExample)
CN (1) CN109863243B (enExample)
AR (1) AR109752A1 (enExample)
AU (1) AU2017333336B2 (enExample)
ES (1) ES2859661T3 (enExample)
PT (1) PT3519569T (enExample)
TW (1) TW201814044A (enExample)
WO (1) WO2018060097A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
US11085055B2 (en) 2014-12-05 2021-08-10 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (fgf21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
MY204731A (en) * 2018-05-30 2024-09-11 Esteve Pharmaceuticals Sa Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
EP3820537A4 (en) * 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
IL283452B2 (en) 2018-11-26 2026-04-01 Univ Barcelona Autonoma FIBROBLAST GROWTH FACTOR 21 (FGF21)-BASED GENE THERAPY
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神(北京)生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
CN114480454B (zh) * 2020-10-27 2024-03-29 华东理工大学 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
CN110496230A (zh) * 2003-02-11 2019-11-26 夏尔人类遗传性治疗公司 使用甲酰-甘氨酸生成酶(fge)对多种硫酸酯酶缺乏症和其它病症进行诊断和治疗
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
SG10201604810UA (en) * 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CN105916990B (zh) * 2013-08-02 2020-02-04 巴塞罗那自治大学 用于转导脂肪组织的腺相关病毒载体
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
GB201322798D0 (en) * 2013-12-20 2014-02-05 Oxford Biomedica Ltd Production system
SG11201609314XA (en) 2014-05-14 2016-12-29 Esteve Labor Dr Adenoassociated virus vectors for the treatment of lysosomal storage disorders
EA201791803A1 (ru) * 2015-02-10 2017-11-30 Джензим Корпорейшн Улучшенная доставка вирусных частиц в полосатое тело и кору больших полушарий
US9981021B1 (en) * 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof

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