BR112015031608A2 - Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos - Google Patents
Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticosInfo
- Publication number
- BR112015031608A2 BR112015031608A2 BR112015031608A BR112015031608A BR112015031608A2 BR 112015031608 A2 BR112015031608 A2 BR 112015031608A2 BR 112015031608 A BR112015031608 A BR 112015031608A BR 112015031608 A BR112015031608 A BR 112015031608A BR 112015031608 A2 BR112015031608 A2 BR 112015031608A2
- Authority
- BR
- Brazil
- Prior art keywords
- vectors
- compositions
- application
- crispr
- systems
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 3
- 210000004185 liver Anatomy 0.000 title abstract 2
- 230000008685 targeting Effects 0.000 title abstract 2
- 238000002560 therapeutic procedure Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 3
- 108091033409 CRISPR Proteins 0.000 abstract 2
- 238000010354 CRISPR gene editing Methods 0.000 abstract 2
- 230000009918 complex formation Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms 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
Classifications
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
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- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
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- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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- A—HUMAN NECESSITIES
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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Abstract
APLICAÇÃO E USO DOS SISTEMAS CRISPR-CAS, VETORES E COMPOSIÇÕES PARA DIRECIONAMENTO E TERAPIA HEPÁTICOS. A invenção provê a aplicação, manipulação e otimização de sistemas, métodos, e composições par a manipulação de sequências e/ou atividades de sequências alvo. São providos sistemas de aplicação e tecidos ou órgãos que são visados como locais de aplicação. São também providos vetores e sistemas de vetores , alguns dos quais codificam um ou mais componentes de um complexo CRISPR, bem como métodos para o desenho e uso desses vetores. Também providos são métodos de direcionamento da formação do complexo CRISPR em células eucarióticas para assegurar especificidade aumentada para reconhecimento do alvo e evitar a toxicidade e para editar ou modificar um local alvo em um local genômico de interesse para alterar ou melhorar o estado de uma doença ou uma afecção.
Applications Claiming Priority (8)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361836123P | 2013-06-17 | 2013-06-17 | |
US201361847537P | 2013-07-17 | 2013-07-17 | |
US201361862355P | 2013-08-05 | 2013-08-05 | |
US201361871301P | 2013-08-28 | 2013-08-28 | |
US201361915325P | 2013-12-12 | 2013-12-12 | |
PCT/US2013/074667 WO2014093622A2 (en) | 2012-12-12 | 2013-12-12 | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
US201461979733P | 2014-04-15 | 2014-04-15 | |
PCT/US2014/041804 WO2014204726A1 (en) | 2013-06-17 | 2014-06-10 | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
Publications (1)
Publication Number | Publication Date |
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BR112015031608A2 true BR112015031608A2 (pt) | 2017-08-22 |
Family
ID=52105121
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112015031608A BR112015031608A2 (pt) | 2013-06-17 | 2014-06-10 | Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos |
Country Status (14)
Country | Link |
---|---|
US (2) | US10577630B2 (pt) |
EP (2) | EP3825406A1 (pt) |
JP (4) | JP6738728B2 (pt) |
KR (1) | KR20160030187A (pt) |
CN (2) | CN107995927B (pt) |
AU (3) | AU2014281028B2 (pt) |
BR (1) | BR112015031608A2 (pt) |
CA (1) | CA2915842C (pt) |
DK (1) | DK3011031T3 (pt) |
IL (1) | IL243196B (pt) |
MX (2) | MX2015017312A (pt) |
RU (1) | RU2716420C2 (pt) |
SG (2) | SG10201710486XA (pt) |
WO (1) | WO2014204726A1 (pt) |
Families Citing this family (342)
Publication number | Priority date | Publication date | Assignee | Title |
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US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
EP3597741A1 (en) | 2012-04-27 | 2020-01-22 | Duke University | Genetic correction of mutated genes |
DK3401400T3 (da) | 2012-05-25 | 2019-06-03 | Univ California | Fremgangsmåder og sammensætninger til rna-styret mål-dna-modifikation og til rna-styret transskriptionsmodulering |
US10323279B2 (en) | 2012-08-14 | 2019-06-18 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US10221442B2 (en) | 2012-08-14 | 2019-03-05 | 10X Genomics, Inc. | Compositions and methods for sample processing |
US20140155295A1 (en) | 2012-08-14 | 2014-06-05 | 10X Technologies, Inc. | Capsule array devices and methods of use |
US11591637B2 (en) | 2012-08-14 | 2023-02-28 | 10X Genomics, Inc. | Compositions and methods for sample processing |
US10273541B2 (en) | 2012-08-14 | 2019-04-30 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US9951386B2 (en) | 2014-06-26 | 2018-04-24 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US10752949B2 (en) | 2012-08-14 | 2020-08-25 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US9701998B2 (en) | 2012-12-14 | 2017-07-11 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US10400280B2 (en) | 2012-08-14 | 2019-09-03 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
CN110643600A (zh) | 2012-10-23 | 2020-01-03 | 基因工具股份有限公司 | 用于切割靶dna的系统及其用途 |
JP6620018B2 (ja) | 2012-12-06 | 2019-12-11 | シグマ−アルドリッチ・カンパニー・リミテッド・ライアビリティ・カンパニーSigma−Aldrich Co., LLC | Crisprに基づくゲノム修飾および制御 |
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 |
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