EA201990212A1 - Композиции на основе системы crispr/cas9 и способы лечения дегенераций сетчатки - Google Patents
Композиции на основе системы crispr/cas9 и способы лечения дегенераций сетчаткиInfo
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- EA201990212A1 EA201990212A1 EA201990212A EA201990212A EA201990212A1 EA 201990212 A1 EA201990212 A1 EA 201990212A1 EA 201990212 A EA201990212 A EA 201990212A EA 201990212 A EA201990212 A EA 201990212A EA 201990212 A1 EA201990212 A1 EA 201990212A1
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- Eurasian Patent Office
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- methods
- crispr
- treatment
- compositions based
- cas9 system
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Abstract
В заявке описаны способы лечения у субъекта дегенерации сетчатки, такой как врожденный амавроз Лебера (LCA), пигментный ретинит (RP) и глаукома. Также здесь предусмотрены способы изменения экспрессии одного или более продуктов генов в клетке, такой как ганглиозная клетка сетчатки. Такие способы могут включать использование модифицированной нуклеазной системы, такой как система коротких палиндромных повторов, регулярно расположенных группами, (CRISPR), включающая двунаправленный промотор H1 и РНК-проводники (gRNA), направленные на гены, связанные с дегенерацией сетчатки, упакованная в одну компактную частицу аденоассоциированного вируса (AAV).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662358337P | 2016-07-05 | 2016-07-05 | |
PCT/US2017/040745 WO2018009562A1 (en) | 2016-07-05 | 2017-07-05 | Crispr/cas9-based compositions and methods for treating retinal degenerations |
Publications (1)
Publication Number | Publication Date |
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EA201990212A1 true EA201990212A1 (ru) | 2020-09-07 |
Family
ID=60913143
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201990212A EA201990212A1 (ru) | 2016-07-05 | 2017-07-05 | Композиции на основе системы crispr/cas9 и способы лечения дегенераций сетчатки |
Country Status (14)
Country | Link |
---|---|
US (1) | US20200080108A1 (ru) |
EP (1) | EP3481434A4 (ru) |
JP (1) | JP2019520391A (ru) |
KR (1) | KR20190039703A (ru) |
CN (1) | CN109890424A (ru) |
AU (1) | AU2017293773A1 (ru) |
BR (1) | BR112019000057A2 (ru) |
CA (1) | CA3029874A1 (ru) |
CL (1) | CL2019000024A1 (ru) |
EA (1) | EA201990212A1 (ru) |
IL (1) | IL264028A (ru) |
MX (1) | MX2019000262A (ru) |
SG (2) | SG10202109385QA (ru) |
WO (1) | WO2018009562A1 (ru) |
Families Citing this family (57)
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WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
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EA039693B1 (ru) * | 2014-06-16 | 2022-02-28 | Дзе Джонс Хопкинс Юниверсити | Композиции и способы для экспрессии рнк-проводников crispr с использованием промотора h1 |
AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
EP3362571A4 (en) | 2015-10-13 | 2019-07-10 | Duke University | GENOMIC ENGINEERING WITH TYPE I CRISPRISMS IN EUKARYOTIC CELLS |
JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 遺伝子編集のための進化したCas9蛋白質 |
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EP3494215A1 (en) | 2016-08-03 | 2019-06-12 | President and Fellows of Harvard College | Adenosine nucleobase editors and uses thereof |
JP7201153B2 (ja) | 2016-08-09 | 2023-01-10 | プレジデント アンド フェローズ オブ ハーバード カレッジ | プログラム可能cas9-リコンビナーゼ融合タンパク質およびその使用 |
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WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
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WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
IL269458B2 (en) | 2017-03-23 | 2024-02-01 | Harvard College | Nucleic base editors that include nucleic acid programmable DNA binding proteins |
US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
US11732274B2 (en) | 2017-07-28 | 2023-08-22 | President And Fellows Of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE) |
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WO2021188993A1 (en) * | 2020-03-20 | 2021-09-23 | Dignity Health | Method of engineering and isolating adeno-associated virus |
IT202000008014A1 (it) | 2020-04-15 | 2021-10-15 | Fond Telethon | RNA guida e loro usi |
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CN111850044A (zh) * | 2020-07-16 | 2020-10-30 | 中国科学技术大学 | 基于在体基因敲除的视网膜色素变性猕猴模型构建方法 |
JP2023549456A (ja) * | 2020-10-21 | 2023-11-27 | デューク ユニバーシティ | デュシェンヌ型筋ジストロフィーの処置のための大きい変異ホットスポットの二重aavベクター媒介欠失 |
WO2023285431A1 (en) | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
KR20230016751A (ko) * | 2021-07-26 | 2023-02-03 | 서울대학교산학협력단 | 염기 편집기 및 이의 용도 |
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CN114550817B (zh) * | 2022-01-25 | 2022-12-23 | 云南大学 | 基于多特征的ctcf介导染色质环预测方法 |
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SG10201804976YA (en) * | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Genome Editing |
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EP3540061A1 (en) * | 2014-04-02 | 2019-09-18 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
EA039693B1 (ru) * | 2014-06-16 | 2022-02-28 | Дзе Джонс Хопкинс Юниверсити | Композиции и способы для экспрессии рнк-проводников crispr с использованием промотора h1 |
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EP4008780A1 (en) * | 2015-04-30 | 2022-06-08 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
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