Academisch Ziekenhuis Leiden

Biomarin Technologies B.v.

Prosensa Technologies B.v.

Other

Academisch Ziekenhuis Leiden (h.o.d.n. Lumc)

Prosena Technologies B.v.

Academisch Ziekenbuls Leiden

Judith Christina Theodora Van Deutekom

Gerard Johannes Platenburg

Josephus Johannes De Kimpe

Garrit-Jan Boudewijn Van Ommen

Annemieke Aartsma-Rus

Peter Christian De Visser

Johannes Theodorus Den Dunnen

Adriana Marie Rus

Petra Van Kuik-Romeijn

Johannes Antonius Heemskerk

Gerardus Johannes Platenburg

Joseph Stephan Verbeek

Aliye Seda Ylmaz-Elis

Nicole Anne Datson

Aliye Seda Yilmaz-Elis

The current invention provides splice-switching compounds with improved characteristics that enhance clinical applicability preferably for treating, ameliorating, preventing, and/or delaying neuromuscular disorders, more specifically DMD.

Bispecific antisense oligonucleotides for dystrophin exon skipping

The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.

Oligonucleotide comprising an inosine for treating DMD

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 an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA

The invention provides means and methods for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy and/or Becker Muscular Dystrophy. Therapies using compounds for providing patients with functional muscle proteins are combined with at least one adjunct compound for reducing inflammation, preferably for reducing muscle tissue inflammation, and/or at least one adjunct compound for improving muscle fiber function, integrity and/or survival.

Means and methods for counteracting muscle disorders

The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing and/or delaying DMD or BMD.

RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy

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 the cell and/or the patient with a molecule. The invention also relates to the molecule as such.

Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53

The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.

Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means

The invention relates to oligonucleotides for inducing skipping of exon 55 of the dystrophin gene. The invention also relates to methods of inducing exon 55 skipping using the oligonucleotides.

Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure

RNA modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy

The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides.

The invention provides a method for generating an oligonucleotide with which an exon may be skipped in a pre-mRNA and thus excluded from a produced mRNA thereof. Further provided are methods for altering the secondary structure of an mRNA to interfere with splicing processes and uses of the oligonucleotides and methods in the treatment of disease. Further provided are pharmaceutical compositions and methods and means for inducing skipping of several exons in a pre-mRNA.

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 isolated 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.

Growing community of inventors

Dordrecht, Netherlands

Judith Christina Theodora Van Deutekom