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1. WO2002024906 - INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS

Publication Number WO/2002/024906
Publication Date 28.03.2002
International Application No. PCT/NL2001/000697
International Filing Date 21.09.2001
Chapter 2 Demand Filed 22.04.2002
IPC
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
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
C12N 15/11 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
11DNA or RNA fragments; Modified forms thereof
CPC
A01K 2267/03
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
A61K 31/7088
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
A61K 48/00
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
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
A61K 9/127
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9Medicinal preparations characterised by special physical form
10Dispersions; Emulsions
127Liposomes
C12N 15/11
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
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
Applicants
  • ACADEMISCH ZIEKENHUIS LEIDEN [NL]/[NL] (AllExceptUS)
  • VAN OMMEN, Garrit-Jan, Boudewijn [NL]/[NL] (UsOnly)
  • VAN DEUTEKOM, Judith, Christina, Theodora [NL]/[NL] (UsOnly)
  • DEN DUNNEN, Johannes, Theodorus [NL]/[NL] (UsOnly)
Inventors
  • VAN OMMEN, Garrit-Jan, Boudewijn
  • VAN DEUTEKOM, Judith, Christina, Theodora
  • DEN DUNNEN, Johannes, Theodorus
Agents
  • PRINS, A., W.
Priority Data
00203283.721.09.2000EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS
(FR) INDUCTION D'OMISSION D'EXON DANS DES CELLULES EUCARYOTES
Abstract
(EN)
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, said cell comprising pre-mRNA comprising exons coding for said protein, by inducing so-called exon skipping in said cell. Exon-skipping results in mature mRNA that does not contain the skipped exon which leads to an altered product if said exon codes for amino acids. Exon skipping is performed by providing a cell with an agent capable of specifically inhibiting an exon inclusion signal, for instance an exon recognition sequence, of said exon. Said exon inclusion signal can be interfered with by a nucleic acid comprising complementarity to a part of said exon. Said nucleic acid, which is also herewith provided, can be used for the preparation of a medicament, for instance for the treatment of an inherited disease.
(FR)
La présente invention concerne une méthode qui permet au moins en partie de diminuer la production d'une protéine aberrante dans une cellule, laquelle cellule comprend un pré-ARNm contenant des exons codant pour ladite protéine, par induction d'une soi-disante omission d'exon dans la cellule. L'omission d'exon débouche sur un ARNm mature exempt de l'exon omis et donne un produit modifié lorsque ledit exon code pour des acides aminés. L'omission d'exon s'effectue par la mise en oeuvre d'une cellule qui comprend un agent pouvant inhiber de manière spécifique un signal d'inclusion d'exon (par exemple une séquence d'identification d'exons) de l'exon. Le signal d'inclusion d'exon peut être brouillé par un acide nucléique complémentaire d'une partie de l'exon. L'acide nucléique, également mis en oeuvre, peut être utilisé pour la préparation d'un médicament destiné, par exemple, au traitement d'une maladie héréditaire.
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