UA118957C2 - Спосіб і композиція для лікування генетичного захворювання - Google Patents
Спосіб і композиція для лікування генетичного захворювання Download PDFInfo
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- UA118957C2 UA118957C2 UAA201501169A UAA201501169A UA118957C2 UA 118957 C2 UA118957 C2 UA 118957C2 UA A201501169 A UAA201501169 A UA A201501169A UA A201501169 A UAA201501169 A UA A201501169A UA 118957 C2 UA118957 C2 UA 118957C2
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- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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| EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| GB201122458D0 (en) | 2011-12-30 | 2012-02-08 | Univ Wageningen | Modified cascade ribonucleoproteins and uses thereof |
| BR112014020625A2 (pt) * | 2012-02-24 | 2017-07-04 | Hutchinson Fred Cancer Res | polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma |
| FI3597749T3 (fi) | 2012-05-25 | 2023-10-09 | Univ California | Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten |
| EP4397760A3 (en) * | 2012-10-23 | 2024-10-09 | Toolgen Incorporated | Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof |
| ES2714154T3 (es) | 2012-12-06 | 2019-05-27 | Sigma Aldrich Co Llc | Modificación y regulación del genoma en base a CRISPR |
| JP6491113B2 (ja) | 2013-02-25 | 2019-03-27 | サンガモ セラピューティクス， インコーポレイテッド | ヌクレアーゼ媒介性遺伝子破壊を増強するための方法および組成物 |
| BR112015022061B8 (pt) | 2013-03-14 | 2023-12-26 | Caribou Biosciences Inc | Ácido nucleico de direcionamento de ácido nucleico guia de fita simples geneticamente manipulado, polinucleotídeo, método para clivar um ácido nucleico alvo e para ligar um ácido nucleico alvo, composição e kit |
| EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| CA2910489A1 (en) * | 2013-05-15 | 2014-11-20 | Sangamo Biosciences, Inc. | Methods and compositions for treatment of a genetic condition |
| AU2014274840B2 (en) * | 2013-06-05 | 2020-03-12 | Duke University | RNA-guided gene editing and gene regulation |
| EP3019595A4 (en) | 2013-07-09 | 2016-11-30 | THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS | |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| WO2015031619A1 (en) * | 2013-08-28 | 2015-03-05 | Sangamo Biosciences, Inc. | Compositions for linking dna-binding domains and cleavage domains |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| JP2016536021A (ja) | 2013-11-07 | 2016-11-24 | エディタス・メディシン，インコーポレイテッド | ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物 |
| DK3068881T3 (en) * | 2013-11-13 | 2019-04-15 | Childrens Medical Center | NUCLEASE-MEDIATED REGULATION OF GENEPRESSION |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| EP3102673B1 (en) * | 2014-02-03 | 2020-04-15 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a beta thalessemia |
| AU2015218576B2 (en) | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
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| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| JP6594891B2 (ja) | 2014-03-18 | 2019-10-23 | サンガモ セラピューティクス， インコーポレイテッド | ジンクフィンガータンパク質発現を調節するための方法および組成物 |
| EP3981876A1 (en) * | 2014-03-26 | 2022-04-13 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| EP3122181A4 (en) * | 2014-03-26 | 2018-04-25 | The Brigham and Women's Hospital, Inc. | Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells |
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| BR112017024115A2 (pt) | 2015-05-12 | 2018-08-07 | Sangamo Therapeutics Inc | regulação de expressão genética mediada por nuclease |
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| US10676735B2 (en) * | 2015-07-22 | 2020-06-09 | Duke University | High-throughput screening of regulatory element function with epigenome editing technologies |
| KR102803519B1 (ko) * | 2015-09-23 | 2025-05-08 | 상가모 테라퓨틱스, 인코포레이티드 | Htt 리프레서 및 이의 용도 |
| KR20180069832A (ko) * | 2015-10-20 | 2018-06-25 | 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 | 유도 cas 시스템을 통한 비기능성 유전자 산물에 대한 기능 회복 및 이용 방법 |
| SG10202104041PA (en) | 2015-10-23 | 2021-06-29 | Harvard College | Nucleobase editors and uses thereof |
| PE20181206A1 (es) | 2015-10-28 | 2018-07-23 | Sangamo Therapeutics Inc | Construcciones especificas del higado, casetes de expresion del factor viii y metodos de uso de estos |
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