Induktion af exon-skipping i eukaryotiske celler

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 contai...

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Hauptverfasser: van Deutekom, Judith Christina Theodora, van Ommen, Garrit-Jan Boudewijn, den Dunnen, Johannes Theodorus
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creator van Deutekom, Judith Christina Theodora
van Ommen, Garrit-Jan Boudewijn
den Dunnen, Johannes Theodorus
description 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.
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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. 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subjects AGRICULTURE
ANIMAL HUSBANDRY
BEER
BIOCHEMISTRY
CARE OF BIRDS, FISHES, INSECTS
CHEMISTRY
COMPOSITIONS OR TEST PAPERS THEREFOR
COMPOSITIONS THEREOF
CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES
CULTURE MEDIA
ENZYMOLOGY
FISHING
FORESTRY
HUMAN NECESSITIES
HUNTING
HYGIENE
MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NEW BREEDS OF ANIMALS
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROCESSES OF PREPARING SUCH COMPOSITIONS
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
TRAPPING
VINEGAR
WINE
title Induktion af exon-skipping i eukaryotiske celler
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