MX2023002978A - Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos. - Google Patents
Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos.Info
- Publication number
- MX2023002978A MX2023002978A MX2023002978A MX2023002978A MX2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A MX 2023002978 A MX2023002978 A MX 2023002978A
- Authority
- MX
- Mexico
- Prior art keywords
- gjb2
- raav
- encoding
- utr
- nucleic acid
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 108010069156 Connexin 26 Proteins 0.000 abstract 9
- 102000055974 Connexin 26 Human genes 0.000 abstract 9
- 102100037156 Gap junction beta-2 protein Human genes 0.000 abstract 2
- 101000954092 Homo sapiens Gap junction beta-2 protein Proteins 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 108020005345 3' Untranslated Regions Proteins 0.000 abstract 1
- 108020003589 5' Untranslated Regions Proteins 0.000 abstract 1
- 206010011878 Deafness Diseases 0.000 abstract 1
- 208000031514 autosomal recessive nonsyndromic hearing loss 1A Diseases 0.000 abstract 1
- 231100000895 deafness Toxicity 0.000 abstract 1
- 210000003027 ear inner Anatomy 0.000 abstract 1
- 239000003623 enhancer Substances 0.000 abstract 1
- 210000000630 fibrocyte Anatomy 0.000 abstract 1
- 208000016354 hearing loss disease Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 201000006790 nonsyndromic deafness Diseases 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 210000002985 organ of corti Anatomy 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
Classifications
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078233P | 2020-09-14 | 2020-09-14 | |
| US202163161619P | 2021-03-16 | 2021-03-16 | |
| PCT/US2021/050205 WO2022056444A1 (en) | 2020-09-14 | 2021-09-14 | Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023002978A true MX2023002978A (es) | 2023-06-01 |
Family
ID=80631939
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023002978A MX2023002978A (es) | 2020-09-14 | 2021-09-14 | Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos. |
Country Status (10)
| Country | Link |
|---|---|
| EP (1) | EP4211151A4 (https=) |
| JP (1) | JP2023541443A (https=) |
| KR (1) | KR20230069157A (https=) |
| AU (1) | AU2021339843A1 (https=) |
| BR (1) | BR112023004605A2 (https=) |
| CA (1) | CA3191533A1 (https=) |
| IL (1) | IL301057A (https=) |
| MX (1) | MX2023002978A (https=) |
| TW (1) | TW202227476A (https=) |
| WO (1) | WO2022056444A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2024011224A2 (en) * | 2022-07-08 | 2024-01-11 | The Trustees Of Columbia University In The City Of New York | Regulatory element for cell type specific expression of genes in spinal motor neurons |
| WO2024218192A1 (en) * | 2023-04-18 | 2024-10-24 | Uniqure Biopharma B.V. | Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs |
| EP4713463A1 (en) * | 2023-05-18 | 2026-03-25 | Decibel Therapeutics, Inc. | Compositions and methods for treating gjb2-related hearing loss |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1308458A1 (en) * | 2001-10-30 | 2003-05-07 | GenOdyssee | New polynucleotides and polypeptides of the CX26 gene |
| DE602005019174D1 (de) * | 2004-06-17 | 2010-03-18 | Epigenomics Ag | Zusammensetzungen und verfahren zur verhinderung des mitschleppens einer kontamination in nukleinsäure-amplifikationsreaktionen |
| CN101389769A (zh) * | 2005-12-28 | 2009-03-18 | 森托科尔公司 | 用于评价和治疗银屑病及相关疾病的标记与方法 |
| WO2019200016A1 (en) * | 2018-04-10 | 2019-10-17 | 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 |
| EP4063510A1 (en) * | 2018-11-07 | 2022-09-28 | Akouos, Inc. | Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear |
| WO2021067448A1 (en) * | 2019-09-30 | 2021-04-08 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of genetic hearing loss |
| WO2021231808A2 (en) * | 2020-05-13 | 2021-11-18 | Akouos, Inc. | Compositions and methods for treating gjb2-associated hearing loss |
| MX2023013394A (es) * | 2021-05-13 | 2023-12-14 | Akouos Inc | Composiciones de suministro de terapia genica y metodos para tratar perdida auditiva. |
-
2021
- 2021-09-14 TW TW110134291A patent/TW202227476A/zh unknown
- 2021-09-14 JP JP2023516689A patent/JP2023541443A/ja active Pending
- 2021-09-14 IL IL301057A patent/IL301057A/en unknown
- 2021-09-14 EP EP21867807.6A patent/EP4211151A4/en active Pending
- 2021-09-14 MX MX2023002978A patent/MX2023002978A/es unknown
- 2021-09-14 BR BR112023004605A patent/BR112023004605A2/pt unknown
- 2021-09-14 WO PCT/US2021/050205 patent/WO2022056444A1/en not_active Ceased
- 2021-09-14 CA CA3191533A patent/CA3191533A1/en active Pending
- 2021-09-14 AU AU2021339843A patent/AU2021339843A1/en active Pending
- 2021-09-14 KR KR1020237012321A patent/KR20230069157A/ko active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3191533A1 (en) | 2022-03-17 |
| EP4211151A1 (en) | 2023-07-19 |
| KR20230069157A (ko) | 2023-05-18 |
| TW202227476A (zh) | 2022-07-16 |
| AU2021339843A1 (en) | 2023-04-06 |
| JP2023541443A (ja) | 2023-10-02 |
| EP4211151A4 (en) | 2025-03-26 |
| IL301057A (en) | 2023-05-01 |
| BR112023004605A2 (pt) | 2023-04-11 |
| WO2022056444A1 (en) | 2022-03-17 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2023002978A (es) | Virus adenoasociado recombinante (raav) que codifica la union gap beta 2 (gjb2) y sus usos. | |
| JP7814280B2 (ja) | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 | |
| Shibata et al. | RNA interference prevents autosomal-dominant hearing loss | |
| Prigione et al. | HIF1 α modulates cell fate reprogramming through early glycolytic shift and upregulation of PDK1–3 and PKM2 | |
| Bellec et al. | CFTR inactivation by lentiviral vector-mediated RNA interference and CRISPR-Cas9 genome editing in human airway epithelial cells | |
| Das et al. | Interferon-inducible protein IFI35 negatively regulates RIG-I antiviral signaling and supports vesicular stomatitis virus replication | |
| WO2022032154A3 (en) | Compositions for the delivery of payload molecules to airway epithelium | |
| PE20211581A1 (es) | Composiciones para la reduccion de la expresion transgenica especifica de drg | |
| CR20210444A (es) | Composiciones y métodos para tratar distrofia cristalina de bietti | |
| Schultz et al. | Differentiation of neurons restricts Arbovirus replication and increases expression of the alpha isoform of IRF-7 | |
| Orimoto et al. | Efficient immortalization of human dental pulp stem cells with expression of cell cycle regulators with the intact chromosomal condition | |
| MX2024008574A (es) | Modulacion de la expresion del virus de la hepatitis b (vhb). | |
| JP2023526053A (ja) | Ｇｊｂ２関連難聴を処置するための組成物および方法 | |
| CL2021000798A1 (es) | Composiciones útiles para el tratamiento de la gangliosidosis gm1. | |
| Aaron et al. | Selection of viral capsids and promoters affects the efficacy of rescue of Tmprss3-deficient cochlea | |
| Song et al. | RBM39 alters phosphorylation of c-Jun and binds to viral RNA to promote PRRSV proliferation | |
| JP2020534810A (ja) | 細胞におけるｒｎａ発現を増強する方法 | |
| PE20240240A1 (es) | Composiciones de administracion de terapia genica y metodos para el tratamiento de la perdida auditiva | |
| Ochs et al. | Post-transcriptional regulation of 5-lipoxygenase mRNA expression via alternative splicing and nonsense-mediated mRNA decay | |
| Duran Alonso et al. | Transcription factor induced conversion of human fibroblasts towards the hair cell lineage | |
| Taiber et al. | Genetic therapies for hearing loss: Accomplishments and remaining challenges | |
| BR112022023908A2 (pt) | Composições de vírus adenoassociados para restauração da função do gene pah e métodos de uso das mesmas | |
| Guo et al. | Specific knockdown of Htra2 by CRISPR-CasRx prevents acquired sensorineural hearing loss in mice | |
| Ghimire et al. | Toll-like receptor 7 (TLR7)-mediated antiviral response protects mice from lethal SARS-CoV-2 infection | |
| Ukaji et al. | AAV-mediated base editing restores cochlear gap junction in GJB2 dominant-negative mutation-associated syndromic hearing loss model |