AR122079A1 - Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4 - Google Patents

Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4

Info

Publication number
AR122079A1
AR122079A1 ARP210101314A ARP210101314A AR122079A1 AR 122079 A1 AR122079 A1 AR 122079A1 AR P210101314 A ARP210101314 A AR P210101314A AR P210101314 A ARP210101314 A AR P210101314A AR 122079 A1 AR122079 A1 AR 122079A1
Authority
AR
Argentina
Prior art keywords
kcnq4
compositions
treating
methods
hearing loss
Prior art date
Application number
ARP210101314A
Other languages
English (en)
Inventor
Emmanuel John Simons
Robert Ng
Linda B Couto
Gregory Scott Robinson
Katherine D Gribble
Original Assignee
Akouos Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Akouos Inc filed Critical Akouos Inc
Publication of AR122079A1 publication Critical patent/AR122079A1/es

Links

Classifications

    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente invención se refiere a tecnologías que comprenden un polinucleótido capaz de expresar y/o inhibir un producto génico de KCNQ4.
ARP210101314A 2020-05-13 2021-05-12 Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4 AR122079A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063024488P 2020-05-13 2020-05-13

Publications (1)

Publication Number Publication Date
AR122079A1 true AR122079A1 (es) 2022-08-10

Family

ID=76584537

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210101314A AR122079A1 (es) 2020-05-13 2021-05-12 Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4

Country Status (5)

Country Link
US (1) US20230212606A1 (es)
EP (1) EP4165194A2 (es)
AR (1) AR122079A1 (es)
TW (1) TW202208406A (es)
WO (1) WO2021231538A2 (es)

Family Cites Families (20)

* Cited by examiner, † Cited by third party
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
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
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
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1399183B1 (en) 2001-05-31 2010-06-30 Novartis Vaccines and Diagnostics, Inc. Chimeric alphavirus replicon particles
AU2002327614B2 (en) 2001-09-06 2007-12-06 Alphavax, Inc. Alphavirus replicon vector systems
NZ600121A (en) 2001-11-13 2013-12-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
SI2292780T1 (sl) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
US7557197B2 (en) 2004-01-28 2009-07-07 Oregon Health & Science University 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
ES2525143T3 (es) 2005-04-07 2014-12-18 The Trustees Of The University Of Pennsylvania Cápsides de AAVrh.64 modificado, composiciones que las contienen y usos de las mismas
EP1712641A1 (en) * 2005-04-14 2006-10-18 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Use of novel HNF4a target genes and their gene products
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
AU2013240248B2 (en) 2012-03-26 2018-12-20 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Delivery of packaged RNA to mammalian cells
WO2019173367A1 (en) * 2018-03-05 2019-09-12 Children's Medical Center Corporation Compositions and methods for delivering nucleic acids to cochlear and vestibular cells

Also Published As

Publication number Publication date
EP4165194A2 (en) 2023-04-19
WO2021231538A2 (en) 2021-11-18
TW202208406A (zh) 2022-03-01
WO2021231538A3 (en) 2022-01-13
US20230212606A1 (en) 2023-07-06

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AR122079A1 (es) Composiciones y métodos para tratar la perdida auditiva asociada a kcnq4