KR102823833B1 - 뮤코다당류증 iiia형에 대한 유전자 요법 - Google Patents

뮤코다당류증 iiia형에 대한 유전자 요법 Download PDF

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KR102823833B1
KR102823833B1 KR1020207018108A KR20207018108A KR102823833B1 KR 102823833 B1 KR102823833 B1 KR 102823833B1 KR 1020207018108 A KR1020207018108 A KR 1020207018108A KR 20207018108 A KR20207018108 A KR 20207018108A KR 102823833 B1 KR102823833 B1 KR 102823833B1
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제임스 엠. 윌슨
네이선 카츠
줄리에트 호르도
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더 트러스티스 오브 더 유니버시티 오브 펜실베니아
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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KR1020207018108A 2017-11-30 2018-11-29 뮤코다당류증 iiia형에 대한 유전자 요법 Active KR102823833B1 (ko)

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Application Number Priority Date Filing Date Title
US201762593081P 2017-11-30 2017-11-30
US62/593,081 2017-11-30
PCT/US2018/063168 WO2019108857A1 (en) 2017-11-30 2018-11-29 Gene therapy for mucopolysaccharidosis iiia

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KR20200104307A KR20200104307A (ko) 2020-09-03
KR102823833B1 true KR102823833B1 (ko) 2025-06-23

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US (2) US11555206B2 (enExample)
EP (1) EP3717653A4 (enExample)
JP (2) JP7389744B2 (enExample)
KR (1) KR102823833B1 (enExample)
AU (1) AU2018375164B2 (enExample)
BR (1) BR112020010735A2 (enExample)
CA (1) CA3083416A1 (enExample)
IL (1) IL274939B1 (enExample)
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US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
JP7389744B2 (ja) * 2017-11-30 2023-11-30 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ｉｉｉａ型のための遺伝子療法
ES3035673T3 (en) * 2018-12-20 2025-09-08 Esteve Pharmaceuticals Sa Recombinant vectors for the long term treatment of mucchopolysacharidosis
CN114746556A (zh) * 2019-07-18 2022-07-12 利索基因公司 用于治疗山菲立普病和其他障碍的组合物和方法
CN115803064A (zh) 2020-05-12 2023-03-14 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
AU2022214429A1 (en) 2021-02-01 2023-09-14 Tern Therapeutics, Llc Gene therapy for neuronal ceroid lipofuscinoses
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒

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IL274939B1 (en) 2025-12-01
KR20200104307A (ko) 2020-09-03
US20200291429A1 (en) 2020-09-17
EP3717653A1 (en) 2020-10-07
AU2018375164A1 (en) 2020-06-04
EP3717653A4 (en) 2021-12-01
JP2023159274A (ja) 2023-10-31
JP2021508241A (ja) 2021-03-04
MX2020005663A (es) 2020-08-20
AU2018375164B2 (en) 2025-05-15
US11555206B2 (en) 2023-01-17
WO2019108857A8 (en) 2020-07-16
JP7389744B2 (ja) 2023-11-30
WO2019108857A1 (en) 2019-06-06
CA3083416A1 (en) 2019-06-06
BR112020010735A2 (pt) 2020-11-10
IL274939A (en) 2020-07-30
US20230279430A1 (en) 2023-09-07

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