METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST EXON 52 OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE

The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecu...

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Hauptverfasser: Platenburg, Gerardus Johannes, van Deutekom, Judith Christina Theodora, van Ommen, Garrit-Jan Boudewijn, de Kimpe, Josephus Johannes, Aartsma-Rus, Annemieke
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creator Platenburg, Gerardus Johannes
van Deutekom, Judith Christina Theodora
van Ommen, Garrit-Jan Boudewijn
de Kimpe, Josephus Johannes
Aartsma-Rus, Annemieke
description The invention relates a method wherein a molecule is used for inducing and/or promoting skipping of at least one of exon 43, exon 46, exons 50-53 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient, the method comprising providing said cell and/or said patient with a molecule. The invention also relates to said molecule as such.
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subjects CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
STEROIDS
title METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST EXON 52 OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE
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