MX2022014085A - Composiciones y metodos para tratar la perdida de audicion asociada a gjb2. - Google Patents
Composiciones y metodos para tratar la perdida de audicion asociada a gjb2.Info
- Publication number
- MX2022014085A MX2022014085A MX2022014085A MX2022014085A MX2022014085A MX 2022014085 A MX2022014085 A MX 2022014085A MX 2022014085 A MX2022014085 A MX 2022014085A MX 2022014085 A MX2022014085 A MX 2022014085A MX 2022014085 A MX2022014085 A MX 2022014085A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- hearing loss
- gjb2
- compositions
- treating
- Prior art date
Links
- 206010011878 Deafness Diseases 0.000 title abstract 3
- 208000016354 hearing loss disease Diseases 0.000 title abstract 3
- 231100000888 hearing loss Toxicity 0.000 title abstract 2
- 230000010370 hearing loss Effects 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title 1
- 108091026890 Coding region Proteins 0.000 abstract 2
- 102000055974 Connexin 26 Human genes 0.000 abstract 1
- 108010069156 Connexin 26 Proteins 0.000 abstract 1
- 231100000895 deafness Toxicity 0.000 abstract 1
Classifications
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- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- 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
- A61K48/005—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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K9/0046—Ear
-
- 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
- A61K9/10—Dispersions; Emulsions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
- A61M25/00—Catheters; Hollow probes
- A61M25/0021—Catheters; Hollow probes characterised by the form of the tubing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
- A61M25/00—Catheters; Hollow probes
- A61M25/0067—Catheters; Hollow probes characterised by the distal end, e.g. tips
- A61M25/0082—Catheter tip comprising a tool
- A61M25/0084—Catheter tip comprising a tool being one or more injection needles
-
- 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
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
-
- 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
- A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
- A61M25/00—Catheters; Hollow probes
- A61M25/0021—Catheters; Hollow probes characterised by the form of the tubing
- A61M2025/0042—Microcatheters, cannula or the like having outside diameters around 1 mm or less
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
- A61M25/00—Catheters; Hollow probes
- A61M25/0067—Catheters; Hollow probes characterised by the distal end, e.g. tips
- A61M25/0082—Catheter tip comprising a tool
- A61M25/0084—Catheter tip comprising a tool being one or more injection needles
- A61M2025/0093—Catheter tip comprising a tool being one or more injection needles wherein at least one needle is a microneedle
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Anesthesiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
- Pulmonology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Inorganic Chemistry (AREA)
Abstract
La presente descripción proporciona construcciones que comprenden una secuencia codificadora enlazada operativamente a un promotor, en donde la secuencia codificadora codifica una proteína de conexina 26. Los ejemplos de construcciones incluyen construcciones de AAV. También se proporcionan métodos para usar construcciones descritas para el tratamiento de pérdida de audición y/o sordera.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063024468P | 2020-05-13 | 2020-05-13 | |
US202163152835P | 2021-02-23 | 2021-02-23 | |
PCT/US2021/032354 WO2021231808A2 (en) | 2020-05-13 | 2021-05-13 | Compositions and methods for treating gjb2-associated hearing loss |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022014085A true MX2022014085A (es) | 2022-12-07 |
Family
ID=78525016
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022014085A MX2022014085A (es) | 2020-05-13 | 2021-05-13 | Composiciones y metodos para tratar la perdida de audicion asociada a gjb2. |
Country Status (11)
Country | Link |
---|---|
US (1) | US20230183743A1 (es) |
EP (1) | EP4164695A4 (es) |
JP (1) | JP2023526053A (es) |
KR (1) | KR20230023641A (es) |
CN (1) | CN116096900A (es) |
AU (1) | AU2021270308A1 (es) |
CA (1) | CA3178197A1 (es) |
CL (1) | CL2022003147A1 (es) |
IL (1) | IL298091A (es) |
MX (1) | MX2022014085A (es) |
WO (1) | WO2021231808A2 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023150689A2 (en) * | 2022-02-04 | 2023-08-10 | Decible Therapeutics, Inc. | Gjb2 regulatory elements and uses thereof |
CN116836975A (zh) * | 2022-03-25 | 2023-10-03 | 上海玮美基因科技有限责任公司 | 一种耳蜗和/或前庭细胞特异性启动子及其应用 |
Family Cites Families (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2005009287A2 (en) * | 2003-07-21 | 2005-02-03 | University Of Maryland, Baltimore | Drug delivery to the inner ear and methods of using same |
CA2919140C (en) * | 2013-07-24 | 2018-05-01 | Commissariat A L'energie Atomique Et Aux Energies Alternatives | Use of flecainide as an anti-connexin agent and method for potentiating the effects of a psychotropic drug |
SG11201804814YA (en) * | 2015-12-11 | 2018-07-30 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
US20210079406A1 (en) * | 2018-04-10 | 2021-03-18 | President And Fellows Of Harvard College | Aav vectors encoding clarin-1 or gjb2 and uses thereof |
WO2020077295A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
US20230056182A1 (en) * | 2018-11-07 | 2023-02-23 | Akouos, Inc. | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
-
2021
- 2021-05-13 EP EP21803692.9A patent/EP4164695A4/en active Pending
- 2021-05-13 KR KR1020227043071A patent/KR20230023641A/ko active Search and Examination
- 2021-05-13 AU AU2021270308A patent/AU2021270308A1/en active Pending
- 2021-05-13 IL IL298091A patent/IL298091A/en unknown
- 2021-05-13 MX MX2022014085A patent/MX2022014085A/es unknown
- 2021-05-13 CA CA3178197A patent/CA3178197A1/en active Pending
- 2021-05-13 US US17/998,486 patent/US20230183743A1/en active Pending
- 2021-05-13 WO PCT/US2021/032354 patent/WO2021231808A2/en unknown
- 2021-05-13 JP JP2022568815A patent/JP2023526053A/ja active Pending
- 2021-05-13 CN CN202180049197.6A patent/CN116096900A/zh active Pending
-
2022
- 2022-11-11 CL CL2022003147A patent/CL2022003147A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
IL298091A (en) | 2023-01-01 |
EP4164695A2 (en) | 2023-04-19 |
WO2021231808A2 (en) | 2021-11-18 |
CN116096900A (zh) | 2023-05-09 |
WO2021231808A3 (en) | 2021-12-23 |
CL2022003147A1 (es) | 2023-07-21 |
KR20230023641A (ko) | 2023-02-17 |
AU2021270308A1 (en) | 2022-12-15 |
CA3178197A1 (en) | 2021-11-18 |
US20230183743A1 (en) | 2023-06-15 |
JP2023526053A (ja) | 2023-06-20 |
EP4164695A4 (en) | 2024-06-05 |
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