CL2022003153A1 - Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 - Google Patents
Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4Info
- Publication number
- CL2022003153A1 CL2022003153A1 CL2022003153A CL2022003153A CL2022003153A1 CL 2022003153 A1 CL2022003153 A1 CL 2022003153A1 CL 2022003153 A CL2022003153 A CL 2022003153A CL 2022003153 A CL2022003153 A CL 2022003153A CL 2022003153 A1 CL2022003153 A1 CL 2022003153A1
- Authority
- CL
- Chile
- Prior art keywords
- methods
- hearing loss
- slc26a4
- 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
- 102000011384 Pendrin Human genes 0.000 abstract 1
- 108050001616 Pendrin Proteins 0.000 abstract 1
- 231100000895 deafness Toxicity 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
- A61K48/005—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
-
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Immunology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Marine Sciences & Fisheries (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente invención se refiere a constructos que comprenden una secuencia codificante enlazada operativamente a un promotor, en donde la secuencia codificante codifica una proteína pendrina. Los constructos ejemplares incluyen constructos de AAV. También se proporcionan métodos de uso de constructos descritos para el tratamiento de la pérdida auditiva y/o sordera.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063024466P | 2020-05-13 | 2020-05-13 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2022003153A1 true CL2022003153A1 (es) | 2023-07-28 |
Family
ID=76584538
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2022003153A CL2022003153A1 (es) | 2020-05-13 | 2022-11-11 | Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 |
Country Status (14)
Country | Link |
---|---|
US (1) | US20230201372A1 (es) |
EP (1) | EP4165195A2 (es) |
JP (1) | JP2023526047A (es) |
KR (1) | KR20230041965A (es) |
CN (1) | CN116801911A (es) |
AR (1) | AR122078A1 (es) |
AU (1) | AU2021272971A1 (es) |
BR (1) | BR112022022906A2 (es) |
CA (1) | CA3183171A1 (es) |
CL (1) | CL2022003153A1 (es) |
IL (1) | IL298128A (es) |
MX (1) | MX2022014204A (es) |
TW (1) | TW202208405A (es) |
WO (1) | WO2021231567A2 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023122720A1 (en) * | 2021-12-23 | 2023-06-29 | University Of Rochester | Compositions and methods for delivery of agents to inner ear |
Family Cites Families (34)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP0173552B1 (en) | 1984-08-24 | 1991-10-09 | The Upjohn Company | Recombinant dna compounds and the expression of polypeptides such as tpa |
US5168062A (en) | 1985-01-30 | 1992-12-01 | University Of Iowa Research Foundation | Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence |
US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
WO1998010088A1 (en) | 1996-09-06 | 1998-03-12 | Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
CA2348382C (en) | 1998-11-10 | 2013-09-17 | The University Of North Carolina At Chapel Hill | Chimeric parvovirus vectors and methods of making and administering the same |
CA2410901C (en) | 2000-06-01 | 2012-03-20 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
CA2448066A1 (en) | 2001-05-31 | 2002-12-12 | Chiron Corporation | Chimeric alphavirus replicon particles |
DE60233061D1 (de) | 2001-09-06 | 2009-09-03 | Alphavax Inc | Alphavirus replikon-vektorsysteme |
CA2756866C (en) | 2001-11-13 | 2017-06-06 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
PL2292780T3 (pl) | 2003-09-30 | 2018-05-30 | The Trustees Of The University Of Pennsylvania | Klady wirusów związanych z adenowirusami (AAV), sekwencje, wektory je zawierające i ich zastosowania |
WO2005073384A2 (en) | 2004-01-28 | 2005-08-11 | Research Development Foundation | Human soluble neuropilin-1 primary polyadenylation signal and uses thereof |
US20060040354A1 (en) | 2004-07-20 | 2006-02-23 | O'keefe Theresa L | Novel polyadenylation signal for use in expression vectors |
ES2580044T3 (es) | 2005-04-07 | 2016-08-18 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
US9506041B2 (en) | 2012-03-26 | 2016-11-29 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Delivery of packaged RNA to mammalian cells |
GB201518979D0 (en) | 2015-10-27 | 2015-12-09 | Univ Leuven Kath | Treatment of hepatic steatosis related oligo-ovulation |
US20190038778A1 (en) * | 2016-02-05 | 2019-02-07 | The General Hospital Corporation | Hybrid System for Efficient Gene Delivery to Cells of the Inner Ear |
CA3034527A1 (en) | 2016-08-23 | 2018-03-01 | Emmanuel John Simons | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
BR112020016919A2 (pt) | 2018-02-22 | 2020-12-15 | Akouos, Inc. | Composições e métodos para tratamento da deficiência auditiva não associada à idade em um sujeito humano |
WO2020077295A1 (en) * | 2018-10-11 | 2020-04-16 | Decibel Therapeutics, Inc. | Aav1 vectors and uses thereof for treatment of otic indications |
-
2021
- 2021-05-12 CA CA3183171A patent/CA3183171A1/en active Pending
- 2021-05-12 AU AU2021272971A patent/AU2021272971A1/en active Pending
- 2021-05-12 EP EP21734252.6A patent/EP4165195A2/en active Pending
- 2021-05-12 IL IL298128A patent/IL298128A/en unknown
- 2021-05-12 AR ARP210101313A patent/AR122078A1/es unknown
- 2021-05-12 TW TW110117159A patent/TW202208405A/zh unknown
- 2021-05-12 KR KR1020227043498A patent/KR20230041965A/ko active Search and Examination
- 2021-05-12 WO PCT/US2021/031983 patent/WO2021231567A2/en active Application Filing
- 2021-05-12 US US17/924,933 patent/US20230201372A1/en active Pending
- 2021-05-12 JP JP2022568802A patent/JP2023526047A/ja active Pending
- 2021-05-12 CN CN202180061214.8A patent/CN116801911A/zh active Pending
- 2021-05-12 BR BR112022022906A patent/BR112022022906A2/pt unknown
- 2021-05-12 MX MX2022014204A patent/MX2022014204A/es unknown
-
2022
- 2022-11-11 CL CL2022003153A patent/CL2022003153A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
BR112022022906A2 (pt) | 2023-01-17 |
KR20230041965A (ko) | 2023-03-27 |
US20230201372A1 (en) | 2023-06-29 |
WO2021231567A3 (en) | 2021-12-23 |
AU2021272971A1 (en) | 2023-02-02 |
AR122078A1 (es) | 2022-08-10 |
JP2023526047A (ja) | 2023-06-20 |
CN116801911A (zh) | 2023-09-22 |
CA3183171A1 (en) | 2021-11-18 |
WO2021231567A2 (en) | 2021-11-18 |
MX2022014204A (es) | 2023-04-14 |
TW202208405A (zh) | 2022-03-01 |
EP4165195A2 (en) | 2023-04-19 |
IL298128A (en) | 2023-01-01 |
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