MX2016007326A - Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. - Google Patents
Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos.Info
- Publication number
- MX2016007326A MX2016007326A MX2016007326A MX2016007326A MX2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A MX 2016007326 A MX2016007326 A MX 2016007326A
- Authority
- MX
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- Prior art keywords
- nucleotide repeat
- methods
- repeat disorders
- crispr
- compositions
- Prior art date
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- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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Abstract
La invención proporciona el suministro, la manipulación y la optimización de sistemas, métodos y composiciones para la manipulación de secuencias y/o actividades de secuencias diana, especialmente para el uso que se refiere a trastornos de repetición de nucleótidos. Se proporcionan sistemas de suministro tejidos u órganos que se escogen como sitios diana para el suministro, especialmente para el uso relativo a trastornos de repetición de nucleótidos. También se proporcionan vectores y sistemas vectoriales, algunos de los cuales codifican uno o más componentes de un complejo o sistema CRISPR, especialmente para el uso relativo a trastornos de repetición de nucleótidos, así como a los métodos para el diseño y de tales. También se proporcionan métodos para dirigir la formación del sistema o complejo CRISPR en células eucariotas especialmente para el uso relativo a trastornos de repetición de nucleótidos incluyendo consideración de especificidad para el reconocimiento de la diana y para evitar toxicidad y editar o modificar un sitio diana en un locus genómico de interés con el fin de alternar o mejorar el estado de una enfermedad o afección.
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361915150P | 2013-12-12 | 2013-12-12 | |
| US201462010879P | 2014-06-11 | 2014-06-11 | |
| US201462010888P | 2014-06-11 | 2014-06-11 | |
| PCT/US2014/069902 WO2015089354A1 (en) | 2013-12-12 | 2014-12-12 | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2016007326A true MX2016007326A (es) | 2017-02-13 |
Family
ID=52273577
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016007325A MX2016007325A (es) | 2013-12-12 | 2014-12-12 | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| MX2016007326A MX2016007326A (es) | 2013-12-12 | 2014-12-12 | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016007325A MX2016007325A (es) | 2013-12-12 | 2014-12-12 | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
Country Status (12)
| Country | Link |
|---|---|
| US (5) | US20160354487A1 (es) |
| EP (4) | EP3080258A1 (es) |
| JP (2) | JP6625055B2 (es) |
| KR (2) | KR20160097338A (es) |
| CN (2) | CN106103705A (es) |
| AU (2) | AU2014362248A1 (es) |
| BR (2) | BR112016013520A2 (es) |
| CA (2) | CA2932472A1 (es) |
| IL (2) | IL246118A0 (es) |
| MX (2) | MX2016007325A (es) |
| SG (2) | SG10201804973TA (es) |
| WO (2) | WO2015089354A1 (es) |
Families Citing this family (354)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| KR102530118B1 (ko) * | 2012-07-25 | 2023-05-08 | 더 브로드 인스티튜트, 인코퍼레이티드 | 유도 dna 결합 단백질 및 게놈 교란 도구 및 이의 적용 |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
| EP2931899A1 (en) | 2012-12-12 | 2015-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| HRP20181648T1 (hr) | 2013-04-16 | 2019-01-25 | Regeneron Pharmaceuticals, Inc. | Ciljana modifikacija genoma štakora |
| WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| WO2014204724A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| JP6738728B2 (ja) | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのCRISPR−Cas系、ベクターおよび組成物の送達および使用 |
| AU2014281031B2 (en) * | 2013-06-17 | 2020-05-21 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components |
| WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| 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 |
| WO2015070083A1 (en) | 2013-11-07 | 2015-05-14 | Editas Medicine,Inc. | CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物 |
| MX2016007325A (es) | 2013-12-12 | 2017-07-19 | Broad Inst Inc | Composiciones y metodos de uso de sistemas crispr-cas en desordenes debidos a repeticion de nucleotidos. |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| JP2017527256A (ja) | 2013-12-12 | 2017-09-21 | ザ・ブロード・インスティテュート・インコーポレイテッド | HBV及びウイルス性疾患及び障害のためのCRISPR−Cas系及び組成物の送達、使用及び治療適用 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| MX374529B (es) | 2013-12-12 | 2025-03-06 | Broad Inst Inc | Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma. |
| CN111705365B (zh) | 2014-02-11 | 2024-12-17 | 科罗拉多州立大学董事会(法人团体) | Crispr支持的多路基因组工程化 |
| WO2015143046A2 (en) * | 2014-03-18 | 2015-09-24 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
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- 2014-12-12 AU AU2014362245A patent/AU2014362245A1/en not_active Abandoned
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- 2014-12-12 EP EP19204059.0A patent/EP3653703A1/en active Pending
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- 2014-12-12 BR BR112016013547A patent/BR112016013547A2/pt not_active Application Discontinuation
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