MX2021004294A - Suministro mejorado de particulas virales al cuerpo estriado y al cortex. - Google Patents
Suministro mejorado de particulas virales al cuerpo estriado y al cortex.Info
- Publication number
- MX2021004294A MX2021004294A MX2021004294A MX2021004294A MX2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A
- Authority
- MX
- Mexico
- Prior art keywords
- striatum
- cortex
- viral particles
- enhanced delivery
- mammal
- Prior art date
Links
- 210000001577 neostriatum Anatomy 0.000 title abstract 3
- 239000002245 particle Substances 0.000 title abstract 3
- 230000003612 virological effect Effects 0.000 title abstract 2
- 241000124008 Mammalia Species 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 210000003169 central nervous system Anatomy 0.000 abstract 1
- 210000003710 cerebral cortex Anatomy 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- 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
- 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
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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
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- 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
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- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biotechnology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Psychology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Abstract
La invención proporciona nuevos métodos para suministrar partículas virales adeno-asociadas recombinantes (rAAV) al sistema nervioso central de un mamífero (p. ej. un ser humano). En aspectos, los métodos comprenden administrar partículas de rAAV que contienen un ácido nucleico heterólogo al cuerpo estriado y causar la expresión del ácido nucleico heterólogo al menos en el córtex cerebral y en el cuerpo estriado del mamífero.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562114544P | 2015-02-10 | 2015-02-10 | |
US201562220997P | 2015-09-19 | 2015-09-19 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021004294A true MX2021004294A (es) | 2021-06-04 |
Family
ID=56614902
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017010370A MX2017010370A (es) | 2015-02-10 | 2016-02-09 | Suministro mejorado de particulas virales al cuerpo estriado y al cortex. |
MX2021004294A MX2021004294A (es) | 2015-02-10 | 2017-08-10 | Suministro mejorado de particulas virales al cuerpo estriado y al cortex. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017010370A MX2017010370A (es) | 2015-02-10 | 2016-02-09 | Suministro mejorado de particulas virales al cuerpo estriado y al cortex. |
Country Status (27)
Country | Link |
---|---|
US (2) | US20190111157A1 (es) |
EP (3) | EP4023758A1 (es) |
JP (3) | JP6928558B2 (es) |
KR (2) | KR20240010539A (es) |
CN (2) | CN107530447A (es) |
AU (2) | AU2016219398A1 (es) |
BR (1) | BR112017017037A2 (es) |
CA (1) | CA2976082A1 (es) |
CL (1) | CL2017002028A1 (es) |
CR (1) | CR20170407A (es) |
DK (1) | DK3256594T3 (es) |
EA (1) | EA201791803A1 (es) |
ES (1) | ES2904504T3 (es) |
HK (1) | HK1246340A1 (es) |
HU (1) | HUE057272T2 (es) |
IL (3) | IL296466A (es) |
LT (1) | LT3256594T (es) |
MX (2) | MX2017010370A (es) |
PH (1) | PH12017501436A1 (es) |
PL (1) | PL3256594T3 (es) |
PT (1) | PT3256594T (es) |
RS (1) | RS62811B1 (es) |
SG (3) | SG10201907399RA (es) |
SI (1) | SI3256594T1 (es) |
TN (1) | TN2017000353A1 (es) |
TW (1) | TWI730952B (es) |
WO (1) | WO2016130591A2 (es) |
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WO2016172155A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
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US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
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US10898585B2 (en) | 2017-04-14 | 2021-01-26 | Ptc Therapeutics .Inc. | Gene therapy for AADC deficiency |
WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
CN110913866A (zh) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
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JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
JP7502991B2 (ja) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症(als)の治療 |
CN110876269B (zh) * | 2018-06-29 | 2021-07-30 | 武汉纽福斯生物科技有限公司 | 治疗遗传性视神经病变的组合物和方法 |
SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
KR20210131370A (ko) * | 2019-02-22 | 2021-11-02 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스 |
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US20220275367A1 (en) * | 2019-07-24 | 2022-09-01 | Voyager Therapeutics, Inc. | Compositions and methods for treating huntington's disease |
WO2021177116A1 (ja) * | 2020-03-02 | 2021-09-10 | 国立大学法人群馬大学 | ミクログリア選択的遺伝子発現ベクター |
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WO2024086747A1 (en) | 2022-10-19 | 2024-04-25 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
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ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
IL292951B1 (en) * | 2014-05-02 | 2024-06-01 | Genzyme Corp | AAV vectors for gene therapy in the central nervous system and retina |
PL3256594T3 (pl) * | 2015-02-10 | 2022-02-14 | Genzyme Corporation | Ulepszone dostarczanie cząstek wirusa do prążkowia i kory |
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2016
- 2016-02-09 PL PL16714066T patent/PL3256594T3/pl unknown
- 2016-02-09 KR KR1020247000976A patent/KR20240010539A/ko active Application Filing
- 2016-02-09 TN TNP/2017/000353A patent/TN2017000353A1/en unknown
- 2016-02-09 CN CN201680020700.4A patent/CN107530447A/zh active Pending
- 2016-02-09 IL IL296466A patent/IL296466A/en unknown
- 2016-02-09 SG SG10201907399RA patent/SG10201907399RA/en unknown
- 2016-02-09 EP EP21198081.8A patent/EP4023758A1/en not_active Withdrawn
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