CA3059112A1 - Cep290 minigene therapy - Google Patents
Cep290 minigene therapy Download PDFInfo
- Publication number
- CA3059112A1 CA3059112A1 CA3059112A CA3059112A CA3059112A1 CA 3059112 A1 CA3059112 A1 CA 3059112A1 CA 3059112 A CA3059112 A CA 3059112A CA 3059112 A CA3059112 A CA 3059112A CA 3059112 A1 CA3059112 A1 CA 3059112A1
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- Prior art keywords
- nucleic acid
- cep290
- isolated nucleic
- raav
- promoter
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| EP3377518B1 (en) * | 2015-11-16 | 2022-04-13 | Synlogic Operating Company, Inc. | Bacteria engineered to reduce hyperphenylalaninemia |
| WO2018187552A1 (en) | 2017-04-05 | 2018-10-11 | University Of Massachusetts | Minigene therapy |
| EP3956452A4 (en) * | 2019-04-19 | 2023-03-15 | University of Massachusetts | GENE THERAPIES FOR USHER SYNDROME (USH2A) |
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| CN114206353B (zh) * | 2019-05-20 | 2024-11-08 | 马萨诸塞大学 | 小基因疗法 |
| JP2022549138A (ja) | 2019-09-18 | 2022-11-24 | インターガラクティック セラピューティクス インコーポレイテッド | 合成ｄｎａベクターおよびその使用法 |
| WO2022023284A1 (en) | 2020-07-27 | 2022-02-03 | Anjarium Biosciences Ag | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| CN112062827B (zh) * | 2020-09-16 | 2022-10-21 | 中国人民解放军军事科学院军事医学研究院 | Cep55蛋白在调控纤毛去组装和制备纤毛相关疾病模型中的应用 |
| WO2022120080A1 (en) * | 2020-12-03 | 2022-06-09 | University Of Massachusetts | Development of novel gene therapeutics for fibrodysplasia ossificans progressiva |
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| EP3393522B1 (en) | 2015-12-22 | 2021-07-07 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Improved hybrid dual recombinant aav vector systems for gene therapy |
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| US20200121746A1 (en) | 2017-06-30 | 2020-04-23 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
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