SG10201804973TA - Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders - Google Patents
Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat DisordersInfo
- Publication number
- SG10201804973TA SG10201804973TA SG10201804973TA SG10201804973TA SG10201804973TA SG 10201804973T A SG10201804973T A SG 10201804973TA SG 10201804973T A SG10201804973T A SG 10201804973TA SG 10201804973T A SG10201804973T A SG 10201804973TA SG 10201804973T A SG10201804973T A SG 10201804973TA
- Authority
- SG
- Singapore
- Prior art keywords
- methods
- nucleotide repeat
- repeat disorders
- compositions
- crispr
- Prior art date
Links
- 239000002773 nucleotide Substances 0.000 title abstract 6
- 238000000034 method Methods 0.000 title abstract 5
- 125000003729 nucleotide group Chemical group 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 6
- 108091033409 CRISPR Proteins 0.000 abstract 2
- 238000010354 CRISPR gene editing Methods 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 230000015572 biosynthetic process 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
Classifications
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0276—Knock-out vertebrates
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- C12N15/102—Mutagenizing nucleic acids
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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- A01K2267/0306—Animal model for genetic diseases
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- B82—NANOTECHNOLOGY
- B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
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- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
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- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
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- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Crystallography & Structural Chemistry (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Environmental Sciences (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Bioinformatics & Computational Biology (AREA)
- Biodiversity & Conservation Biology (AREA)
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Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361915150P | 2013-12-12 | 2013-12-12 | |
US201462010888P | 2014-06-11 | 2014-06-11 | |
US201462010879P | 2014-06-11 | 2014-06-11 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG10201804973TA true SG10201804973TA (en) | 2018-07-30 |
Family
ID=52273577
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201804973TA SG10201804973TA (en) | 2013-12-12 | 2014-12-12 | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
SG10201804974RA SG10201804974RA (en) | 2013-12-12 | 2014-12-12 | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201804974RA SG10201804974RA (en) | 2013-12-12 | 2014-12-12 | Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders |
Country Status (12)
Country | Link |
---|---|
US (5) | US20160354487A1 (ja) |
EP (4) | EP3653704A1 (ja) |
JP (2) | JP6712948B2 (ja) |
KR (2) | KR20160097338A (ja) |
CN (2) | CN106029880A (ja) |
AU (2) | AU2014362245A1 (ja) |
BR (2) | BR112016013520A2 (ja) |
CA (2) | CA2932436A1 (ja) |
IL (2) | IL246116B (ja) |
MX (2) | MX2016007325A (ja) |
SG (2) | SG10201804973TA (ja) |
WO (2) | WO2015089351A1 (ja) |
Families Citing this family (283)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
EP3808844A1 (en) * | 2012-07-25 | 2021-04-21 | The Broad Institute, Inc. | Inducible dna binding proteins and genome perturbation tools and applications thereof |
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 |
EP2931897B1 (en) | 2012-12-12 | 2017-11-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
SG11201508028QA (en) | 2013-04-16 | 2015-10-29 | Regeneron Pharma | Targeted modification of rat genome |
CN107995927B (zh) | 2013-06-17 | 2021-07-30 | 布罗德研究所有限公司 | 用于肝靶向和治疗的crispr-cas系统、载体和组合物的递送与用途 |
CN106062197A (zh) | 2013-06-17 | 2016-10-26 | 布罗德研究所有限公司 | 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化 |
KR20160056869A (ko) * | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
EP3725885A1 (en) | 2013-06-17 | 2020-10-21 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
AU2014281027A1 (en) | 2013-06-17 | 2016-01-28 | Massachusetts Institute Of Technology | Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation |
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 |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
KR102380245B1 (ko) | 2013-11-07 | 2022-03-30 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
CN105980568B (zh) | 2013-12-11 | 2019-12-03 | 瑞泽恩制药公司 | 用于靶向修饰基因组的方法和组合物 |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
CA2932478A1 (en) | 2013-12-12 | 2015-06-18 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
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 |
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. |
KR102496984B1 (ko) | 2014-02-11 | 2023-02-06 | 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 | Crispr 이용의 다중화된 게놈 조작 |
US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
CA2942762C (en) * | 2014-03-18 | 2023-10-17 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of zinc finger protein expression |
WO2015173436A1 (en) * | 2014-05-16 | 2015-11-19 | Vrije Universiteit Brussel | Genetic correction of myotonic dystrophy type 1 |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
US10435685B2 (en) * | 2014-08-19 | 2019-10-08 | Pacific Biosciences Of California, Inc. | Compositions and methods for enrichment of nucleic acids |
CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
WO2016073990A2 (en) | 2014-11-07 | 2016-05-12 | Editas Medicine, Inc. | Methods for improving crispr/cas-mediated genome-editing |
US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
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