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1. US20160159846 - Methods and compounds useful in conditions related to repeat expansion

Office États-Unis d'Amérique
Numéro de la demande 14908429
Date de la demande 31.07.2014
Numéro de publication 20160159846
Date de publication 09.06.2016
Numéro de délivrance 10435430
Date de délivrance 08.10.2019
Type de publication B2
CIB
C12N 15/113
CCHIMIE; MÉTALLURGIE
12BIOCHIMIE; BIÈRE; SPIRITUEUX; VIN; VINAIGRE; MICROBIOLOGIE; ENZYMOLOGIE; TECHNIQUES DE MUTATION OU DE GÉNÉTIQUE
NMICRO-ORGANISMES OU ENZYMES; COMPOSITIONS LES CONTENANT; CULTURE OU CONSERVATION DE MICRO-ORGANISMES; TECHNIQUES DE MUTATION OU DE GÉNÉTIQUE; MILIEUX DE CULTURE
15Techniques de mutation ou génie génétique; ADN ou ARN concernant le génie génétique, vecteurs, p.ex. plasmides, ou leur isolement, leur préparation ou leur purification; Utilisation d'hôtes pour ceux-ci
09Technologie d'ADN recombinant
11Fragments d'ADN ou d'ARN; Leurs formes modifiées
113Acides nucléiques non codants modulant l'expression des gènes, p.ex. oligonucléotides anti-sens
C07H 21/02
CCHIMIE; MÉTALLURGIE
07CHIMIE ORGANIQUE
HSUCRES; LEURS DÉRIVÉS; NUCLÉOSIDES; NUCLÉOTIDES; ACIDES NUCLÉIQUES
21Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p.ex. acides nucléiques
02avec le ribosyle comme radical saccharide
CPC
C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C07H 21/02
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02with ribosyl as saccharide radical
C12N 2310/11
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
11Antisense
C12N 2310/32
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
2310Structure or type of the nucleic acid
30Chemical structure
32of the sugar
C12N 2320/34
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
2320Applications; Uses
30Special therapeutic applications
34Allele or polymorphism specific uses
Déposants Ionis Pharmaceuticals, Inc.
Board of Regents of the University of Texas System
Inventeurs Thazha P. Prakash
Jiaxin Hu
Jing Liu
Dongbo Yu
David Corey
Eric E. Swayze
Mandataires Parker Highlander PLLC
Titre
(EN) Methods and compounds useful in conditions related to repeat expansion
Abrégé
(EN)

Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.

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