CA2882499C - Cells genetically modified within the bcl11a gene by a nuclease and various aspects related thereto - Google Patents
Cells genetically modified within the bcl11a gene by a nuclease and various aspects related thereto Download PDFInfo
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| NZ728024A (en) | 2012-05-25 | 2019-05-31 | Univ California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| DE202013012597U1 (de) * | 2012-10-23 | 2017-11-21 | Toolgen, Inc. | Zusammensetzung zum Spalten einer Ziel-DNA, umfassend eine für die Ziel-DNA spezifische guide-RNA und eine Cas-Protein-codierende Nukleinsäure oder ein Cas-Protein, sowie deren Verwendung |
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| CA2901676C (en) | 2013-02-25 | 2023-08-22 | Sangamo Biosciences, Inc. | Methods and compositions for enhancing nuclease-mediated gene disruption |
| US9260752B1 (en) | 2013-03-14 | 2016-02-16 | Caribou Biosciences, Inc. | Compositions and methods of nucleic acid-targeting nucleic acids |
| EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
| EP3730615A3 (en) | 2013-05-15 | 2020-12-09 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
| JP7085716B2 (ja) * | 2013-06-05 | 2022-06-17 | デューク ユニバーシティ | Ｒｎａガイド遺伝子編集及び遺伝子調節 |
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| US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
| EP4372090A3 (en) | 2013-11-07 | 2024-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions with governing grnas |
| DK3068881T3 (en) | 2013-11-13 | 2019-04-15 | Childrens Medical Center | NUCLEASE-MEDIATED REGULATION OF GENEPRESSION |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| ES2796087T3 (es) * | 2014-02-03 | 2020-11-25 | Sangamo Therapeutics Inc | Métodos y composiciones para el tratamiento de beta talasemia |
| US10370680B2 (en) | 2014-02-24 | 2019-08-06 | Sangamo Therapeutics, Inc. | Method of treating factor IX deficiency using nuclease-mediated targeted integration |
| US11028388B2 (en) | 2014-03-05 | 2021-06-08 | Editas Medicine, Inc. | CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa |
| US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| WO2015138510A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine., Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
| US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
| WO2015143046A2 (en) | 2014-03-18 | 2015-09-24 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
| EP3981876A1 (en) * | 2014-03-26 | 2022-04-13 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
| CN112063586A (zh) * | 2014-03-26 | 2020-12-11 | 布里格姆及妇女医院股份有限公司 | 用于人造血干/祖细胞的离体扩增的组合物和方法 |
| WO2015148860A1 (en) * | 2014-03-26 | 2015-10-01 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating beta-thalassemia |
| EP4079847A1 (en) | 2014-07-30 | 2022-10-26 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| AU2015308910B2 (en) | 2014-08-27 | 2017-12-07 | Caribou Biosciences, Inc. | Methods for increasing Cas9-mediated engineering efficiency |
| DK3194570T3 (da) * | 2014-09-16 | 2021-09-13 | Sangamo Therapeutics Inc | Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller |
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