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
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JP2020526514A5 (enExample
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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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JP2020526514A5 JP2020526514A5 (enExample) 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 (enExample)
EP (1) EP3676385A1 (enExample)
JP (2) JP2020526514A (enExample)
KR (2) KR20240063170A (enExample)
AU (1) AU2018298133A1 (enExample)
BR (1) BR112020000063A2 (enExample)
CA (1) CA3068328A1 (enExample)
IL (1) IL271780A (enExample)
MA (1) MA50016A (enExample)
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EP3747998B1 (en) 2013-03-15 2024-07-24 The Trustees of the University of Pennsylvania Compositions for treating mpsi
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
US10973929B2 (en) 2016-02-03 2021-04-13 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type I
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
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
BR112021009739A2 (pt) * 2018-11-30 2021-10-19 Novartis Ag Vetores virais aav e usos dos mesmos
CA3155154A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
JP2023511382A (ja) * 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
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
KR20230066360A (ko) * 2020-08-10 2023-05-15 프리베일 테라퓨틱스, 인크. 신경퇴행성 장애를 위한 유전자 요법
EP4284335A1 (en) 2021-02-01 2023-12-06 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
WO2022178419A1 (en) * 2021-02-22 2022-08-25 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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