MX2019002518A - Metodos y vectores para el tratamiento de trastornos del snc. - Google Patents
Metodos y vectores para el tratamiento de trastornos del snc.Info
- Publication number
- MX2019002518A MX2019002518A MX2019002518A MX2019002518A MX2019002518A MX 2019002518 A MX2019002518 A MX 2019002518A MX 2019002518 A MX2019002518 A MX 2019002518A MX 2019002518 A MX2019002518 A MX 2019002518A MX 2019002518 A MX2019002518 A MX 2019002518A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- vectors
- cns disorders
- treating cns
- mammalian
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 3
- 239000013598 vector Substances 0.000 title 1
- 210000003169 central nervous system Anatomy 0.000 abstract 3
- 210000000056 organ Anatomy 0.000 abstract 3
- 241000124008 Mammalia Species 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 210000004556 brain Anatomy 0.000 abstract 1
Classifications
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/775—Apolipopeptides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01031—Beta-glucuronidase (3.2.1.31)
-
- 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
-
- 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
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0312—Animal model for Alzheimer's disease
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
- C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Ophthalmology & Optometry (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Dermatology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662383274P | 2016-09-02 | 2016-09-02 | |
| PCT/US2017/049959 WO2018045347A1 (en) | 2016-09-02 | 2017-09-01 | Methods and vectors for treating cns disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019002518A true MX2019002518A (es) | 2019-07-18 |
Family
ID=61309371
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2019002518A MX2019002518A (es) | 2016-09-02 | 2017-09-01 | Metodos y vectores para el tratamiento de trastornos del snc. |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20190192693A1 (enExample) |
| EP (1) | EP3506930A4 (enExample) |
| JP (2) | JP7610923B2 (enExample) |
| CN (1) | CN110121356A (enExample) |
| AU (2) | AU2017318717B2 (enExample) |
| BR (1) | BR112019004353A2 (enExample) |
| CA (1) | CA3035628A1 (enExample) |
| MX (1) | MX2019002518A (enExample) |
| WO (1) | WO2018045347A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| MX2020000713A (es) * | 2017-07-17 | 2020-08-31 | Spark Therapeutics Inc | Metodos de aferesis y sus usos. |
| US12275948B2 (en) | 2018-06-28 | 2025-04-15 | The University Of North Carolina At Chapel Hill | Optimized CLN5 genes and expression cassettes and their use |
| JP7624724B2 (ja) | 2018-09-26 | 2025-01-31 | カリフォルニア インスティテュート オブ テクノロジー | 標的遺伝子療法のためのアデノ随伴ウイルス組成物 |
| JP2022513034A (ja) * | 2018-11-14 | 2022-02-07 | レジェンクスバイオ インコーポレーテッド | 神経セロイドリポフスチン症の遺伝子治療 |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| JP7637058B2 (ja) | 2019-02-22 | 2025-02-27 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| US20220348638A1 (en) * | 2019-09-24 | 2022-11-03 | The J. David Gladstone Institutes, a testamentary trust estabilished under the Will of J. David Glad | Method of targeting neuronal apoe to treat a neurocognitive disorder |
| MX2022004524A (es) * | 2019-10-16 | 2022-07-21 | Univ Cornell | Tratamiento génico para la enfermedad de alzheimer. |
| AU2021351720A1 (en) * | 2020-10-01 | 2023-05-11 | Sangamo Therapeutics, Inc. | Methods of detecting anti-aav antibodies |
| CN112852881B (zh) * | 2021-02-09 | 2023-08-15 | 南京贝思奥生物科技有限公司 | 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法 |
| TW202426479A (zh) * | 2022-10-10 | 2024-07-01 | 美商星火治療公司 | Apoe基因療法 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7135339B2 (en) * | 2003-11-20 | 2006-11-14 | University Of Iowa Research Foundation | Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV) |
| JP5704361B2 (ja) * | 2010-10-27 | 2015-04-22 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| IN2014KN02672A (enExample) * | 2012-05-18 | 2015-05-08 | Univ Iowa Res Found | |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| CA2931220A1 (en) * | 2013-11-20 | 2015-05-28 | University Of Iowa Research Foundation | Methods and compositions for treating amyloid deposits |
| AU2016219789B2 (en) * | 2015-02-20 | 2021-03-25 | Fondazione Telethon | Methods and compositions for treating genetic eye diseases |
-
2017
- 2017-09-01 CN CN201780067677.9A patent/CN110121356A/zh active Pending
- 2017-09-01 WO PCT/US2017/049959 patent/WO2018045347A1/en not_active Ceased
- 2017-09-01 JP JP2019512292A patent/JP7610923B2/ja active Active
- 2017-09-01 AU AU2017318717A patent/AU2017318717B2/en active Active
- 2017-09-01 EP EP17847658.6A patent/EP3506930A4/en active Pending
- 2017-09-01 MX MX2019002518A patent/MX2019002518A/es unknown
- 2017-09-01 US US16/329,697 patent/US20190192693A1/en not_active Abandoned
- 2017-09-01 CA CA3035628A patent/CA3035628A1/en active Pending
- 2017-09-01 BR BR112019004353A patent/BR112019004353A2/pt unknown
-
2022
- 2022-01-11 JP JP2022002163A patent/JP2022050574A/ja active Pending
-
2024
- 2024-10-06 AU AU2024227138A patent/AU2024227138A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022050574A (ja) | 2022-03-30 |
| AU2017318717A1 (en) | 2019-03-21 |
| JP7610923B2 (ja) | 2025-01-09 |
| CA3035628A1 (en) | 2018-03-08 |
| WO2018045347A1 (en) | 2018-03-08 |
| EP3506930A4 (en) | 2020-09-23 |
| CN110121356A (zh) | 2019-08-13 |
| AU2017318717B2 (en) | 2024-10-24 |
| US20190192693A1 (en) | 2019-06-27 |
| JP2019529385A (ja) | 2019-10-17 |
| EP3506930A1 (en) | 2019-07-10 |
| BR112019004353A2 (pt) | 2019-05-28 |
| AU2024227138A1 (en) | 2024-10-31 |
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