JP7449223B2 - ムコ多糖症ｉｉ型を治療するための遺伝子療法 - Google Patents

ムコ多糖症ｉｉ型を治療するための遺伝子療法 Download PDF

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JP7449223B2
JP7449223B2 JP2020516820A JP2020516820A JP7449223B2 JP 7449223 B2 JP7449223 B2 JP 7449223B2 JP 2020516820 A JP2020516820 A JP 2020516820A JP 2020516820 A JP2020516820 A JP 2020516820A JP 7449223 B2 JP7449223 B2 JP 7449223B2
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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JP2020516820A 2017-09-22 2018-09-21 ムコ多糖症ｉｉ型を治療するための遺伝子療法 Active JP7449223B2 (ja)

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JP2024031557A JP2024063154A (ja) 2017-09-22 2024-03-01 ムコ多糖症ｉｉ型を治療するための遺伝子療法

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US201762561769P 2017-09-22 2017-09-22
US201762562179P 2017-09-22 2017-09-22
US62/562,179 2017-09-22
US62/561,769 2017-09-22
US201762573841P 2017-10-18 2017-10-18
US62/573,841 2017-10-18
PCT/US2018/052129 WO2019060662A1 (en) 2017-09-22 2018-09-21 GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II

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JP2024031557A Pending JP2024063154A (ja) 2017-09-22 2024-03-01 ムコ多糖症ｉｉ型を治療するための遺伝子療法

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EP (1) EP3684938A1 (enExample)
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KR (2) KR102883778B1 (enExample)
AU (1) AU2018335752B2 (enExample)
BR (1) BR112020005249A2 (enExample)
CA (1) CA3076036A1 (enExample)
IL (1) IL273427B2 (enExample)
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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
JP7684296B2 (ja) * 2019-11-22 2025-05-27 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 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
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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
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CA3209611A1 (en) * 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
US20240366789A1 (en) * 2021-04-01 2024-11-07 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
CN115820740A (zh) * 2021-09-16 2023-03-21 北京锦篮基因科技有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用
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