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1. WO2020097002 - OPTIMIZED FIG4 GENES AND EXPRESSION CASSETTES AND THEIR USE

Publication Number WO/2020/097002
Publication Date 14.05.2020
International Application No. PCT/US2019/059752
International Filing Date 05.11.2019
IPC
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
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
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
A61P 21/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
21Drugs for disorders of the muscular or neuromuscular system
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
  • THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL [US]/[US]
Inventors
  • GRAY, Steven James
  • BAILEY, Rachel
Agents
  • MYERS BIGEL, P.A.
Priority Data
62/755,87105.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) OPTIMIZED FIG4 GENES AND EXPRESSION CASSETTES AND THEIR USE
(FR) GÈNES FIG4 OPTIMISÉS ET CASSETTES D'EXPRESSION ET LEUR UTILISATION
Abstract
(EN)
This invention relates to polynucleotides comprising optimized FIG4 open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a FIG4 gene or aberrant activity of a FIG4 gene product in the subject, such as CMT4J.
(FR)
La présente invention concerne des polynucléotides comprenant des séquences de cadres de lecture ouverts (ORF) FIG4 optimisés, des vecteurs les comprenant, et des procédés d'utilisation de ceux-ci pour l'administration des ORF à une cellule ou à un sujet et pour traiter des troubles associés à une expression aberrante d'un gène FIG4 ou d'une activité aberrante d'un produit génique FIG4 chez le sujet, tel que CMT4J.
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