BR112016013547A2 - Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos - Google Patents
Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeosInfo
- Publication number
- BR112016013547A2 BR112016013547A2 BR112016013547A BR112016013547A BR112016013547A2 BR 112016013547 A2 BR112016013547 A2 BR 112016013547A2 BR 112016013547 A BR112016013547 A BR 112016013547A BR 112016013547 A BR112016013547 A BR 112016013547A BR 112016013547 A2 BR112016013547 A2 BR 112016013547A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- crispr
- compositions
- systems
- nucleotide repeat
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 3
- 239000002773 nucleotide Substances 0.000 title abstract 2
- 239000013598 vector Substances 0.000 abstract 3
- 108091033409 CRISPR Proteins 0.000 abstract 2
- 238000010354 CRISPR gene editing Methods 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 230000009918 complex formation Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 210000003527 eukaryotic cell Anatomy 0.000 abstract 1
- 238000005457 optimization Methods 0.000 abstract 1
- 210000000056 organ Anatomy 0.000 abstract 1
- 230000001988 toxicity Effects 0.000 abstract 1
- 231100000419 toxicity Toxicity 0.000 abstract 1
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Abstract
COMPOSIÇÕES E MÉTODOS DE USO DE SISTEMAS CRISPR-CAS EM DISTÚRBIOS DE REPETIÇÕES DE NUCLEOTÍDEOS. A invenção proporciona a administração, manipulação e otimização de sistemas, métodos, e composições para manipulação de sequências e/ou atividades de sequências alvo. São proporcionados sistemas de administração e tecidos ou órgãos que são visados como locais de administração. Também são proporcionados vetores e sistemas de vetores, alguns dos quais codificam um ou mais componentes de um complexo CRISPR SIN, bem como métodos para o planejamento e uso de tais vetores. Também são proporcionados métodos de direcionamento da formação do complexo CRISPR SIN em células eucarióticas para assegurar maior especificidade para reconhecimento de alvos e evitar a toxicidade e para editar ou modificar um local alvo em um lócus genômico de interesse, para alterar ou melhorar o estado de uma doença ou de um estado clínico.
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/US2014/069897 WO2015089351A1 (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 |
---|---|
BR112016013547A2 true BR112016013547A2 (pt) | 2017-10-03 |
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Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
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BR112016013520A BR112016013520A2 (pt) | 2013-12-12 | 2014-12-12 | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
BR112016013547A BR112016013547A2 (pt) | 2013-12-12 | 2014-12-12 | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
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BR112016013520A BR112016013520A2 (pt) | 2013-12-12 | 2014-12-12 | Composições e métodos de uso de sistemas crispr-cas em distúrbios de repetições de nucleotídeos |
Country Status (12)
Country | Link |
---|---|
US (5) | US20160354487A1 (pt) |
EP (4) | EP3080257A1 (pt) |
JP (2) | JP6625055B2 (pt) |
KR (2) | KR20160097338A (pt) |
CN (2) | CN106029880A (pt) |
AU (2) | AU2014362248A1 (pt) |
BR (2) | BR112016013520A2 (pt) |
CA (2) | CA2932472A1 (pt) |
IL (2) | IL246116B (pt) |
MX (2) | MX2016007326A (pt) |
SG (2) | SG10201804973TA (pt) |
WO (2) | WO2015089354A1 (pt) |
Families Citing this family (285)
Publication number | Priority date | Publication date | Assignee | Title |
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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 |
JP2015527889A (ja) * | 2012-07-25 | 2015-09-24 | ザ ブロード インスティテュート, インコーポレイテッド | 誘導可能なdna結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用 |
MX2015007550A (es) | 2012-12-12 | 2017-02-02 | Broad Inst Inc | Suministro, modificación y optimización de sistemas, métodos y composiciones para la manipulación de secuencias y aplicaciones terapéuticas. |
WO2014093709A1 (en) | 2012-12-12 | 2014-06-19 | 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 |
CN111500630A (zh) | 2013-04-16 | 2020-08-07 | 瑞泽恩制药公司 | 大鼠基因组的靶向修饰 |
EP3011029B1 (en) | 2013-06-17 | 2019-12-11 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
AU2014281028B2 (en) | 2013-06-17 | 2020-09-10 | Massachusetts Institute Of Technology | Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy |
EP4245853A3 (en) | 2013-06-17 | 2023-10-18 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
JP6702858B2 (ja) | 2013-06-17 | 2020-06-03 | ザ・ブロード・インスティテュート・インコーポレイテッド | ウイルス成分を使用して障害および疾患をターゲティングするためのCRISPR−Cas系および組成物の送達、使用および治療上の適用 |
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 |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9834791B2 (en) | 2013-11-07 | 2017-12-05 | Editas Medicine, Inc. | CRISPR-related methods and compositions with governing gRNAS |
US9546384B2 (en) | 2013-12-11 | 2017-01-17 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a mouse genome |
EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
CN111206032A (zh) | 2013-12-12 | 2020-05-29 | 布罗德研究所有限公司 | 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用 |
SG10201804973TA (en) | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
MX2016010285A (es) | 2014-02-11 | 2017-01-11 | Univ Colorado Regents | Ingenieria genetica multiplexada habilitada por crispr. |
CN106459894B (zh) * | 2014-03-18 | 2020-02-18 | 桑格摩生物科学股份有限公司 | 用于调控锌指蛋白表达的方法和组合物 |
WO2015143078A1 (en) | 2014-03-18 | 2015-09-24 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
WO2015173436A1 (en) * | 2014-05-16 | 2015-11-19 | Vrije Universiteit Brussel | Genetic correction of myotonic dystrophy type 1 |
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