Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
Inventors:
Josephus Johannes De Kimpe, Annemieke Aartsma-Rus, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, Garrit-Jan Boudewijn Van Ommen
Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
Inventors:
Josephus Johannes De Kimpe, Annemieke Aartsma-Rus, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, Garrit-Jan Boudewijn Van Ommen
Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing an isolate muscle cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in the method.
Inventors:
Josephus Johannes De Kimpe, Annemieke Aartsma-Rus, Gerard Johannes Platenburg, Judith Christina Theodora Van Deutekom, Garrit-Jan Boudewijn Van Ommen
Abstract: The invention relates to glycoside-compound conjugates for use in antisense strategies and/or gene therapy. The conjugates comprise a glycoside linked to a compound, in which the glycoside is a ligand capable of binding to a mannose-6-phosphate receptor of a muscle cell. For example the cells are muscle cells of a Duchenne Muscular Dystrophy (DMD) patient and the conjugate comprises an antisense oligonucleotide which causes ex on skipping and induces or restores the synthesis of dystrophin or variants thereof.
Abstract: The invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with a molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such molecule used in said method.
Inventors:
JOSEPHUS JOHANNES DE KIMPE, GERARDUS JOHANNES PLATENBURG, JUDITH CHRISTINA THEODORA VAN DEUTEKOM, ANNEMIEKE AARTSMA-RUS, GARRIT-JAN BOUDEWIJN VAN OMMEN
Abstract: 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.
Inventors:
Josephus Johannes De Kimpe, Gerardus Johannes Platenburg, Judith Christina Theodora Van Deutekom, Annemieke Aartsma-Rus, Garrit-Jan Boudewijn Van Ommen
Abstract: The invention relates to glycoside-compound conjugates for use in antisense strategies and/or gene therapy. The conjugates comprise a glycoside linked to a compound, in which the glycoside is a ligand capable of binding to a mannose-6-phosphate receptor of a muscle cell. For example the cells are muscle cells of a Duchenne Muscular Dystrophy (DMD) patient and the conjugate comprises an antisense oligonucleotide which causes exon skipping and induces or restores the synthesis of dystrophin or variants thereof.
Abstract: Methods for treating neuroblastoma in a subject are provided. The methods include administering to the subject nucleic acid that is capable of causing a significant reduction of the amount of doublecortin like protein (DCL) in the subject. The nucleic acids include antisense oligonucleotides and small interfering RNA.
Type:
Application
Filed:
May 24, 2010
Publication date:
January 27, 2011
Applicants:
PROSENSA B.V., Universiteit Leiden
Inventors:
Erno VREUGDENHIL, Petra Van Kuik-Romeijn
Abstract: The invention relates to glycoside-compound conjugates for use in antisense strategies and/or gene therapy. The conjugates comprise a glycoside linked to a compound, in which the glycoside is a ligand capable of binding to a mannose-6-phosphate receptor of a muscle cell. For example the cells are muscle cells of a Duchenne Muscular Dystrophy (DMD) patient and the conjugate comprises an antisense oligonucleotide which causes exon skipping and induces or restores the synthesis of dystrophin or variants thereof.