JP2020526514A - ムコ多糖症ｉ型を治療するための遺伝子治療 - Google Patents

ムコ多糖症ｉ型を治療するための遺伝子治療 Download PDF

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JP2020526514A
JP2020526514A JP2020500131A JP2020500131A JP2020526514A JP 2020526514 A JP2020526514 A JP 2020526514A JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020526514 A JP2020526514 A JP 2020526514A
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immunosuppressant
hidua
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JP2020526514A5 (https=
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ヒンデラー，クリスチャン
ウイルソン，ジェームス・エム
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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
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JP2020500131A 2017-07-06 2018-07-05 ムコ多糖症ｉ型を治療するための遺伝子治療 Pending JP2020526514A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023108011A JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症ｉ型を治療するための遺伝子治療

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762529385P 2017-07-06 2017-07-06
US62/529,385 2017-07-06
US201762530614P 2017-07-10 2017-07-10
US62/530,614 2017-07-10
US201862616106P 2018-01-11 2018-01-11
US62/616,106 2018-01-11
PCT/US2018/040957 WO2019010335A1 (en) 2017-07-06 2018-07-05 AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I

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JP2020526514A true JP2020526514A (ja) 2020-08-31
JP2020526514A5 JP2020526514A5 (https=) 2021-08-12

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JP2020500131A Pending JP2020526514A (ja) 2017-07-06 2018-07-05 ムコ多糖症ｉ型を治療するための遺伝子治療
JP2023108011A Active JP7670759B2 (ja) 2017-07-06 2023-06-30 ムコ多糖症ｉ型を治療するための遺伝子治療

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US (2) US11890329B2 (https=)
EP (1) EP3676385A1 (https=)
JP (2) JP2020526514A (https=)
KR (2) KR20240063170A (https=)
AU (1) AU2018298133A1 (https=)
BR (1) BR112020000063A2 (https=)
CA (1) CA3068328A1 (https=)
IL (1) IL271780A (https=)
MA (1) MA50016A (https=)
SG (1) SG11201912631PA (https=)
WO (1) WO2019010335A1 (https=)

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CN120860258A (zh) * 2018-11-30 2025-10-31 北京诺华制药有限公司 Aav病毒载体及其用途
WO2021081217A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
KR20220131522A (ko) * 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
AU2021325891A1 (en) * 2020-08-10 2023-04-06 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
JP2023537903A (ja) * 2020-08-10 2023-09-06 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害に対する遺伝子療法
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022170082A1 (en) * 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
EP4294461A4 (en) * 2021-02-22 2025-06-25 Decibel Therapeutics, Inc. METHODS AND COMPOSITIONS FOR REDUCING NUCLEIC ACID VECTOR-INDUCED TOXICITY IN THE INNER EAR
WO2024205528A1 (en) * 2023-03-31 2024-10-03 Erci̇yes Üni̇versi̇tesi̇ Strateji̇ Geli̇şti̇rme Dai̇re Başkanliği A multi-head for use in electrohydrodynamic

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