MX2016006106A - Metodos y composiciones para tratar la enfermedad de huntington. - Google Patents
Metodos y composiciones para tratar la enfermedad de huntington.Info
- Publication number
- MX2016006106A MX2016006106A MX2016006106A MX2016006106A MX2016006106A MX 2016006106 A MX2016006106 A MX 2016006106A MX 2016006106 A MX2016006106 A MX 2016006106A MX 2016006106 A MX2016006106 A MX 2016006106A MX 2016006106 A MX2016006106 A MX 2016006106A
- Authority
- MX
- Mexico
- Prior art keywords
- compositions
- disease
- methods
- huntington
- treating huntington
- Prior art date
Links
- 208000023105 Huntington disease Diseases 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title abstract 4
- 108700028369 Alleles Proteins 0.000 abstract 2
- 108010073062 Transcription Activator-Like Effectors Proteins 0.000 abstract 2
- 108091033409 CRISPR Proteins 0.000 abstract 1
- 238000010354 CRISPR gene editing Methods 0.000 abstract 1
- 230000004568 DNA-binding Effects 0.000 abstract 1
- 101710185494 Zinc finger protein Proteins 0.000 abstract 1
- 102100023597 Zinc finger protein 816 Human genes 0.000 abstract 1
- 238000010171 animal model Methods 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- 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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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6811—Selection methods for production or design of target specific oligonucleotides or binding molecules
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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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/10—Peptides having 12 to 20 amino acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/164—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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
- C12N15/113—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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- C12N15/00—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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- Analytical Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Resumen: En la presente se describen métodos y composiciones para tratar la enfermedad de Huntington. En particular, se proporcionan en la presente métodos y composiciones para modificar (por ejemplo, modular la expresión de) un alelo HD de htt para tratar la enfermedad de Huntington. También se proporcionan métodos y composiciones para generar modelos animales de la enfermedad de Huntington. De este modo, en un aspecto, se proporcionan dominios de unión al ADN diseñados, es decir, que no ocurren de manera natural, (por ejemplo: proteínas de dedo de zinc, proteínas de efector TAL [TALE, por sus siglas en inglés] o CRISPR/dCas-TF) que modulan la expresión de un alelo HD, por ejemplo, htt.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361902704P | 2013-11-11 | 2013-11-11 | |
PCT/US2014/064987 WO2015070212A1 (en) | 2013-11-11 | 2014-11-11 | Methods and compositions for treating huntington's disease |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2016006106A true MX2016006106A (es) | 2016-12-09 |
Family
ID=53042231
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016006106A MX2016006106A (es) | 2013-11-11 | 2014-11-11 | Metodos y composiciones para tratar la enfermedad de huntington. |
Country Status (12)
Country | Link |
---|---|
US (1) | US10369201B2 (es) |
EP (1) | EP3068905A4 (es) |
JP (2) | JP2016537341A (es) |
KR (1) | KR102431079B1 (es) |
CN (1) | CN105934524A (es) |
AU (1) | AU2014346424B2 (es) |
BR (1) | BR112016010175A2 (es) |
CA (1) | CA2929179A1 (es) |
MX (1) | MX2016006106A (es) |
NZ (1) | NZ719477A (es) |
RU (1) | RU2693891C1 (es) |
WO (1) | WO2015070212A1 (es) |
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AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
KR101835401B1 (ko) | 2012-07-13 | 2018-03-08 | 신 니뽄 바이오메디칼 라보라토리즈, 엘티디. | 키랄 핵산 어쥬번트 |
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EP3068905A1 (en) | 2016-09-21 |
WO2015070212A1 (en) | 2015-05-14 |
JP2016537341A (ja) | 2016-12-01 |
RU2693891C1 (ru) | 2019-07-05 |
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AU2014346424B2 (en) | 2020-09-17 |
KR20160077208A (ko) | 2016-07-01 |
RU2016123086A (ru) | 2017-12-19 |
EP3068905A4 (en) | 2017-07-05 |
KR102431079B1 (ko) | 2022-08-11 |
NZ719477A (en) | 2022-05-27 |
BR112016010175A2 (pt) | 2017-10-03 |
US20160296605A1 (en) | 2016-10-13 |
CN105934524A (zh) | 2016-09-07 |
AU2014346424A1 (en) | 2016-05-19 |
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