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1. WO2020097121 - ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN EXCITATORY CORTICAL NEURONS

Publication Number WO/2020/097121
Publication Date 14.05.2020
International Application No. PCT/US2019/059927
International Filing Date 05.11.2019
IPC
C12N 15/67 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
67General methods for enhancing the expression
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 5/0793 2010.01
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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
079Neural cells
0793Neurons
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants
  • ALLEN INSTITUTE [US]/[US]
Inventors
  • GRAYBUCK, Lucas T.
  • TASIC, Bosiljka
  • DAIGLE, Tanya
  • TING, Jonathan
  • ZENG, Hongkui
  • KALMBACH, Brian Edward
  • MICH, John K.
  • HESS, Erik
  • LEIN, Edward Sebastian
  • LEVI, Boaz P.
Agents
  • WINGER, C. Rachal
  • NGUYEN, Thu
Priority Data
62/755,98805.11.2018US
62/806,60015.02.2019US
62/806,68415.02.2019US
62/872,02109.07.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN EXCITATORY CORTICAL NEURONS
(FR) CONSTRUCTIONS D'EXPRESSION ARTIFICIELLE POUR MODULER SÉLECTIVEMENT L'EXPRESSION GÉNIQUE DANS DES NEURONES CORTICAUX EXCITATEURS
Abstract
(EN)
Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in excitatory cortical neurons, such as primarily within cortical layers 2/3, 4, 5, and 6 and including those with extratelencephalic (ET) projections, intratelencephalic (IT) projections, and pyramidal tract (PT) projections, among others.
(FR)
L'invention concerne des constructions d'expression artificielle pour moduler sélectivement l'expression génique dans des types cellulaires du système nerveux central sélectionnés. Les constructions d'expression artificielle peuvent être utilisées pour exprimer sélectivement des gènes synthétiques ou modifier l'expression génique dans des neurones corticaux excitateurs, tels que principalement dans les couches corticales 2/3, 4, 5 et 6 et comprenant celles ayant des projections extratélencéphaliques (ET), des projections intratélencéphaliques (IT) et des projections des voies pyramidales (PT), entre autres.
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