CN114450031A - 靶向rna的敲低和替代组合物及使用方法 - Google Patents
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| EP4216964A4 (en) * | 2020-09-24 | 2025-02-12 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing apoe expression |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2022221278A1 (en) * | 2021-04-12 | 2022-10-20 | Locanabio, Inc. | Compositions and methods comprising hybrid promoters |
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| EP4493702A4 (en) * | 2022-03-18 | 2026-04-01 | Univ Florida | METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
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| WO2025043118A1 (en) * | 2023-08-23 | 2025-02-27 | Ohio State Innovation Foundation | Making tiny rnas inside the cell |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140249212A1 (en) * | 2011-09-27 | 2014-09-04 | Yale University | Compositions and Methods for Transient Expression of Recombinant RNA |
| WO2015164773A1 (en) * | 2014-04-25 | 2015-10-29 | Shire Human Genetic Therapies, Inc. | Methods for purification of messenger rna |
| CN105087650A (zh) * | 2007-10-05 | 2015-11-25 | 吉尼松公司 | 使用外周注射aav载体向运动神经元进行广泛基因投送 |
| CN108690861A (zh) * | 2017-04-10 | 2018-10-23 | 肖晓莺 | 用于早期乳腺癌症诊断的微小核糖核酸 |
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| CN101895633A (zh) | 2010-07-14 | 2010-11-24 | 中兴通讯股份有限公司 | 一种移动终端及其解锁方法 |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| DK2784157T3 (da) | 2011-10-21 | 2019-10-21 | Univ Kyushu Nat Univ Corp | Designfremgangsmåde til et rna-bindende protein under anvendelse af ppr-motiv og anvendelse deraf |
| US10330674B2 (en) | 2015-01-13 | 2019-06-25 | Massachusetts Institute Of Technology | Pumilio domain-based modular protein architecture for RNA binding |
| EP3858992A1 (en) * | 2015-03-13 | 2021-08-04 | The Jackson Laboratory | A three-component crispr/cas complex system and uses thereof |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| US10876101B2 (en) | 2017-03-28 | 2020-12-29 | Locanabio, Inc. | CRISPR-associated (Cas) protein |
| US10392616B2 (en) | 2017-06-30 | 2019-08-27 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
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Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN105087650A (zh) * | 2007-10-05 | 2015-11-25 | 吉尼松公司 | 使用外周注射aav载体向运动神经元进行广泛基因投送 |
| US20140249212A1 (en) * | 2011-09-27 | 2014-09-04 | Yale University | Compositions and Methods for Transient Expression of Recombinant RNA |
| WO2015164773A1 (en) * | 2014-04-25 | 2015-10-29 | Shire Human Genetic Therapies, Inc. | Methods for purification of messenger rna |
| CN106164248A (zh) * | 2014-04-25 | 2016-11-23 | 夏尔人类遗传性治疗公司 | 信使rna的纯化方法 |
| CN108690861A (zh) * | 2017-04-10 | 2018-10-23 | 肖晓莺 | 用于早期乳腺癌症诊断的微小核糖核酸 |
Non-Patent Citations (3)
| Title |
|---|
| YI-TING TSAI等: "Clustered Regularly lnterspaced Short Palindromic Repeats-based genome surgery for the treatment of autosomal dominantretinitis pigmentosa", OPHTHALMOLOGY, vol. 125, no. 9, 11 May 2018 (2018-05-11) * |
| 凌宏艳;廖端芳;: "微小RNA研究进展", 中国动脉硬化杂志, no. 05, 26 May 2008 (2008-05-26) * |
| 陈如意;陈素峰;周丹;许健;: "2型腺相关病毒作为基因治疗载体的研究进展", 生命科学, no. 06, 15 June 2013 (2013-06-15) * |
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| EP3997227A1 (en) | 2022-05-18 |
| US20210009987A1 (en) | 2021-01-14 |
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