JP2016512683A5 - - Google Patents

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JP2016512683A5
JP2016512683A5 JP2016501864A JP2016501864A JP2016512683A5 JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5 JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5
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Japan
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seq
raav
enhancer
change
hidua
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JP2016501864A
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Japanese (ja)
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JP2016512683A (ja
JP6591956B2 (ja
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Priority claimed from PCT/US2014/025509 external-priority patent/WO2014151341A1/en
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JP2016501864A 2013-03-15 2014-03-13 Mps1を治療するための組成物および方法 Active JP6591956B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361788724P 2013-03-15 2013-03-15
US61/788,724 2013-03-15
PCT/US2014/025509 WO2014151341A1 (en) 2013-03-15 2014-03-13 Compositions and methods for treating mpsi

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019079881A Division JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2016512683A JP2016512683A (ja) 2016-05-09
JP2016512683A5 true JP2016512683A5 (enExample) 2017-04-13
JP6591956B2 JP6591956B2 (ja) 2019-10-16

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ID=51580934

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2016501864A Active JP6591956B2 (ja) 2013-03-15 2014-03-13 Mps1を治療するための組成物および方法
JP2019079881A Pending JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法
JP2020148103A Active JP7168621B2 (ja) 2013-03-15 2020-09-03 Mps1を治療するための組成物および方法
JP2022172314A Active JP7553527B2 (ja) 2013-03-15 2022-10-27 Mps1を治療するための組成物および方法

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2019079881A Pending JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法
JP2020148103A Active JP7168621B2 (ja) 2013-03-15 2020-09-03 Mps1を治療するための組成物および方法
JP2022172314A Active JP7553527B2 (ja) 2013-03-15 2022-10-27 Mps1を治療するための組成物および方法

Country Status (21)

Country Link
US (3) US10137176B2 (enExample)
EP (2) EP3747998B1 (enExample)
JP (4) JP6591956B2 (enExample)
KR (2) KR102346455B1 (enExample)
CN (1) CN105026554B (enExample)
AU (2) AU2014235096B2 (enExample)
BR (1) BR112015021036A2 (enExample)
CA (1) CA2901328C (enExample)
CY (1) CY1123169T1 (enExample)
DK (1) DK2984166T3 (enExample)
ES (2) ES2805355T3 (enExample)
HR (1) HRP20201063T1 (enExample)
HU (1) HUE051373T2 (enExample)
LT (1) LT2984166T (enExample)
MX (2) MX386059B (enExample)
PL (1) PL2984166T3 (enExample)
PT (1) PT2984166T (enExample)
RU (1) RU2708318C2 (enExample)
SG (2) SG11201507507PA (enExample)
SI (1) SI2984166T1 (enExample)
WO (1) WO2014151341A1 (enExample)

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TW202229560A (zh) 2020-10-09 2022-08-01 賓州大學委員會 治療法布瑞氏症之組成物及方法
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CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
JP2025512969A (ja) 2022-04-06 2025-04-22 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Her2陽性転移性乳がん及び他のがんを治療するための組成物及び方法
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WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
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JP2020526514A (ja) 2017-07-06 2020-08-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症i型を治療するための遺伝子治療

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