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1. WO2021091950 - VESTIBULAR SUPPORTING CELL PROMOTERS AND USES THEREOF

Publication Number WO/2021/091950
Publication Date 14.05.2021
International Application No. PCT/US2020/058795
International Filing Date 04.11.2020
IPC
A61K 31/52 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
519ortho- or peri-condensed with heterocyclic rings
52Purines, e.g. adenine
A61K 45/06 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
45Medicinal preparations containing active ingredients not provided for in groups A61K31/-A61K41/132
06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
A61K 35/30 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
C12N 15/85 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
C12N 15/86 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
A61P 25/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25Drugs for disorders of the nervous system
Applicants
  • DECIBEL THERAPEUTICS, INC. [US]/[US]
Inventors
  • BURNS, Joseph
  • GIBSON, Tyler
  • PREGERNIG, Gabriela
Agents
  • BELLIVEAU, Michael, J.
Priority Data
62/930,52004.11.2019US
63/024,95914.05.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) VESTIBULAR SUPPORTING CELL PROMOTERS AND USES THEREOF
(FR) PROMOTEURS DE CELLULES DE SUPPORT VESTIBULAIRES ET LEURS UTILISATIONS
Abstract
(EN)
The disclosure provides polynucleotides containing SLC6A14 promoters, as well as vectors containing the same, that can be used to promote expression of a transgene in vestibular supporting cells. The polynucleotides described herein may be operably linked to a transgene, such as a transgene encoding a therapeutic protein, so as to promote vestibular supporting cell expression of the transgene. The polynucleotides described herein may be operably linked to a therapeutic transgene and used for the treatment of subjects having or at risk of developing vestibular dysfunction.
(FR)
La présente invention concerne des polynucléotides contenant des promoteurs SLC6A14, ainsi que des vecteurs contenant ceux-ci, qui peuvent être utilisés pour favoriser l'expression d'un transgène dans des cellules de support vestibulaires. Les polynucléotides selon la présente invention peuvent être liés de manière fonctionnelle à un transgène, tel qu'un transgène codant pour une protéine thérapeutique, de sorte à favoriser l'expression de la cellule de support vestibulaire du transgène. Les polynucléotides selon la présente invention peuvent être liés de manière fonctionnelle à un transgène thérapeutique et utilisés pour le traitement de sujets présentant ou susceptibles de développer un dysfonctionnement vestibulaire.
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