CL2016003247A1 - Composiciones y métodos para la expresión de arns guía usando el promotor h1 - Google Patents
Composiciones y métodos para la expresión de arns guía usando el promotor h1Info
- Publication number
- CL2016003247A1 CL2016003247A1 CL2016003247A CL2016003247A CL2016003247A1 CL 2016003247 A1 CL2016003247 A1 CL 2016003247A1 CL 2016003247 A CL2016003247 A CL 2016003247A CL 2016003247 A CL2016003247 A CL 2016003247A CL 2016003247 A1 CL2016003247 A1 CL 2016003247A1
- Authority
- CL
- Chile
- Prior art keywords
- expression
- promoter
- methods
- compositions
- arng
- Prior art date
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
- C12N15/1138—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 against receptors or cell surface proteins
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- C12N15/90—Stable introduction of foreign DNA into chromosome
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- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
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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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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
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Abstract
<p>LA MATERIA OBJETO DE LA PRESENTE DIVULGACIÓN PROPORCIONA COMPOSICIONES Y MÉTODOS PARA LA EXPRESIÓN DE ARNs GUÍAS DE CRISPR UTILIZANDO EL PROMOTOR H1. EN PARTICULAR, SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA EL USO DEL PROMOTOR H1 PARA EXPRESAR UN ARN GUÍA (ARNg) DE CRISPR CON ESPECIFICIDAD ALTERADA DEL NUCLEÓTIDO EN 5', ASÍ COMO EL USO DE LA SECUENCIA PROMOTORA H1 COMO UN PROMOTOR BIDIRECCIONAL PARA EXPRESAR LA NUCLEASA Cas9 Y EL ARNg SIMULTÁNEAMENTE. TAMBIÉN SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA LA EXPRESIÓN Y LA REGULACIÓN DE LA EXPRESIÓN ARNg IN VIVO MEDIANTE EL USO DE RIBOZIMAS DE ARN Y APTAZIMAS REGULABLES.</p>
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
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US201462012802P | 2014-06-16 | 2014-06-16 |
Publications (1)
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CL2016003247A1 true CL2016003247A1 (es) | 2017-11-24 |
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CL2016003247A CL2016003247A1 (es) | 2014-06-16 | 2016-12-16 | Composiciones y métodos para la expresión de arns guía usando el promotor h1 |
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Country | Link |
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US (5) | US9907863B2 (es) |
EP (2) | EP3155101B1 (es) |
JP (2) | JP6930834B2 (es) |
KR (1) | KR102649341B1 (es) |
CN (2) | CN106852157B (es) |
AU (2) | AU2015277369B2 (es) |
BR (1) | BR112016029178A2 (es) |
CA (1) | CA2952697A1 (es) |
CL (1) | CL2016003247A1 (es) |
DK (1) | DK3155101T3 (es) |
EA (1) | EA039693B1 (es) |
ES (1) | ES2788426T3 (es) |
IL (2) | IL249584B (es) |
MX (2) | MX2016016833A (es) |
SG (2) | SG11201610405RA (es) |
WO (1) | WO2015195621A1 (es) |
Families Citing this family (108)
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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 |
CA2908697C (en) | 2013-04-16 | 2023-12-12 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
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 |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
CA2948728A1 (en) | 2014-03-10 | 2015-09-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10) |
US11339437B2 (en) | 2014-03-10 | 2022-05-24 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
US11141493B2 (en) | 2014-03-10 | 2021-10-12 | Editas Medicine, Inc. | Compositions and methods for treating CEP290-associated disease |
US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
EP3122880B1 (en) | 2014-03-26 | 2021-05-05 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating sickle cell disease |
WO2015195621A1 (en) | 2014-06-16 | 2015-12-23 | The Johns Hopkins University | Compositions and methods for the expression of crispr guide rnas using the h1 promoter |
AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
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- 2015-11-24 US US14/951,240 patent/US9907863B2/en active Active
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2016
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2018
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2019
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2020
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2021
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2022
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