KR20220003553A - 레트 증후군의 치료에 유용한 조성물 - Google Patents
레트 증후군의 치료에 유용한 조성물 Download PDFInfo
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- KR20220003553A KR20220003553A KR1020217037510A KR20217037510A KR20220003553A KR 20220003553 A KR20220003553 A KR 20220003553A KR 1020217037510 A KR1020217037510 A KR 1020217037510A KR 20217037510 A KR20217037510 A KR 20217037510A KR 20220003553 A KR20220003553 A KR 20220003553A
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| PCT/US2020/029642 WO2020219766A1 (en) | 2019-04-24 | 2020-04-23 | Compositions useful in treatment of rett syndrome |
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| WO2022094078A1 (en) * | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| AU2022369293A1 (en) * | 2021-10-18 | 2024-05-16 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| US20250312486A1 (en) * | 2022-04-18 | 2025-10-09 | City Of Hope | Compositions for restoring mecp2 gene function and methods of use thereof |
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| EP4715058A1 (en) * | 2023-05-15 | 2026-03-25 | Genecombio Ltd. | Gene therapy for rett syndrome |
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| PL2002003T3 (pl) * | 2005-05-27 | 2016-06-30 | Ospedale San Raffaele Srl | Wektor genetyczny zawierający mi-RNA |
| PL2010563T3 (pl) * | 2006-04-07 | 2011-11-30 | Georg August Univ Goettingen Stiftung Oeffentlichen Rechts | Sekwencja syntetycznego MECP2 do białkowej terapii substytucyjnej |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| AU2013243948A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| EP3452101A2 (en) * | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| CA3052487A1 (en) * | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Influenza vaccines based on aav vectors |
| JP7455579B2 (ja) * | 2017-02-28 | 2024-03-26 | 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 | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| JP2020513831A (ja) * | 2017-03-24 | 2020-05-21 | ザ ユニバーシティ コート オブ ザ ユニバーシティ オブ エジンバラ | ＭｅＣＰ２発現カセット |
| US11680275B2 (en) * | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
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