SG11201510327TA - Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells - Google Patents
Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cellsInfo
- Publication number
- SG11201510327TA SG11201510327TA SG11201510327TA SG11201510327TA SG11201510327TA SG 11201510327T A SG11201510327T A SG 11201510327TA SG 11201510327T A SG11201510327T A SG 11201510327TA SG 11201510327T A SG11201510327T A SG 11201510327TA SG 11201510327T A SG11201510327T A SG 11201510327TA
- Authority
- SG
- Singapore
- Prior art keywords
- targeting
- disorders
- optimization
- engineering
- delivery
- Prior art date
Links
- 208000037765 diseases and disorders Diseases 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 230000000394 mitotic effect Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 238000005457 optimization Methods 0.000 title 1
- 230000008685 targeting Effects 0.000 title 1
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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
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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
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
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- C12N9/22—Ribonucleases RNAses, DNAses
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
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- C12N2750/14011—Parvoviridae
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- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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- C12N2750/14011—Parvoviridae
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- C12N2750/14151—Methods of production or purification of viral material
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- Zoology (AREA)
- Biomedical Technology (AREA)
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- Medicinal Chemistry (AREA)
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- Bioinformatics & Computational Biology (AREA)
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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 | |
US201361915203P | 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 |
US201461979573P | 2014-04-15 | 2014-04-15 | |
PCT/US2014/041808 WO2014204728A1 (en) | 2013-06-17 | 2014-06-11 | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201510327TA true SG11201510327TA (en) | 2016-01-28 |
Family
ID=52105123
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201510327TA SG11201510327TA (en) | 2013-06-17 | 2014-06-11 | Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells |
SG10201710488TA SG10201710488TA (en) | 2013-06-17 | 2014-06-11 | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Targeting and Modeling Diseases and Disorders of Post Mitotic Cells |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201710488TA SG10201710488TA (en) | 2013-06-17 | 2014-06-11 | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Targeting and Modeling Diseases and Disorders of Post Mitotic Cells |
Country Status (15)
Country | Link |
---|---|
US (1) | US20160153004A1 (en) |
EP (2) | EP3620524A1 (en) |
JP (2) | JP6738729B2 (en) |
KR (1) | KR20160019553A (en) |
CN (1) | CN105683379A (en) |
AU (1) | AU2014281030B2 (en) |
BR (1) | BR112015031611A2 (en) |
CA (1) | CA2915845A1 (en) |
DK (1) | DK3011032T3 (en) |
ES (1) | ES2767318T3 (en) |
IL (1) | IL243197B (en) |
MX (1) | MX2015017311A (en) |
RU (1) | RU2725502C2 (en) |
SG (2) | SG11201510327TA (en) |
WO (1) | WO2014204728A1 (en) |
Families Citing this family (240)
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US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
WO2013139861A1 (en) | 2012-03-20 | 2013-09-26 | Luc Montagnier | Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders |
EP3597741A1 (en) | 2012-04-27 | 2020-01-22 | Duke University | Genetic correction of mutated genes |
EP4289948A3 (en) | 2012-05-25 | 2024-04-17 | The Regents of the University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
JP6517143B2 (en) | 2012-10-23 | 2019-05-22 | ツールゲン インコーポレイテッド | Composition for cleaving target DNA comprising guide RNA specific for target DNA and CAS protein encoding nucleic acid or CAS protein, and use thereof |
US20160017366A1 (en) | 2012-12-06 | 2016-01-21 | Sigma-Aldrich Co. Llc | Crispr-based genome modification and regulation |
EP2931892B1 (en) | 2012-12-12 | 2018-09-12 | 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 |
US20140179770A1 (en) | 2012-12-12 | 2014-06-26 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
HUE040575T2 (en) | 2013-04-16 | 2019-03-28 | Regeneron Pharma | Targeted modification of rat genome |
US10704060B2 (en) | 2013-06-05 | 2020-07-07 | Duke University | RNA-guided gene editing and gene regulation |
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 |
KR20160034901A (en) | 2013-06-17 | 2016-03-30 | 더 브로드 인스티튜트, 인코퍼레이티드 | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
CA2915842C (en) * | 2013-06-17 | 2022-11-29 | The Broad Institute, Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
KR20160056869A (en) | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
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 |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
ES2844174T3 (en) | 2013-09-18 | 2021-07-21 | Kymab Ltd | Methods, cells and organisms |
CN106459995B (en) | 2013-11-07 | 2020-02-21 | 爱迪塔斯医药有限公司 | CRISPR-associated methods and compositions using dominant grnas |
AU2014360811B2 (en) | 2013-12-11 | 2017-05-18 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for the targeted modification of a genome |
EP3080258A1 (en) | 2013-12-12 | 2016-10-19 | The Broad Institute, Inc. | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
CN106061510B (en) * | 2013-12-12 | 2020-02-14 | 布罗德研究所有限公司 | Delivery, use and therapeutic applications of CRISPR-CAS systems and compositions for genome editing |
US20150166985A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting von willebrand factor point mutations |
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 |
AU2015222944A1 (en) | 2014-02-27 | 2016-09-08 | Massachusetts Institute Of Technology | T cell balance gene expression, compositions of matters and methods of use thereof |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
EP3180426B1 (en) | 2014-08-17 | 2019-12-25 | The Broad Institute, Inc. | Genome editing using cas9 nickases |
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EP3626832A3 (en) | 2014-11-25 | 2020-05-13 | The Brigham and Women's Hospital, Inc. | Method of identifying and treating a person having a predisposition to or afflicted with a cardiometabolic disease |
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JP6830437B2 (en) | 2014-12-10 | 2021-02-17 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | Genetically modified cells, tissues and organs to treat the disease |
WO2016094872A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Dead guides for crispr transcription factors |
WO2016094880A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs) |
WO2016094874A1 (en) | 2014-12-12 | 2016-06-16 | The Broad Institute Inc. | Escorted and functionalized guides for crispr-cas systems |
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WO2016100974A1 (en) | 2014-12-19 | 2016-06-23 | The Broad Institute Inc. | Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing |
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BR112015031611A2 (en) | 2017-12-12 |
JP2020063238A (en) | 2020-04-23 |
WO2014204728A1 (en) | 2014-12-24 |
CN105683379A (en) | 2016-06-15 |
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