Proqr Therapeutics Ii B.v.

Wings Therapeutics, Inc.

Academisch Ziekenhuis Leiden

Biomarin Technologies B.v.

Prosensa Holding Bv

Prosensa Technologies B.v.

Prosensa B.v.

Prosensa Holding N.v.

Gerardus Johannes Platenburg

Josephus Johannes De Kimpe

Bart Klein

Marko Potman

Elisabeth Marlene Haisma

Judith Christina Theodora Van Deutekom

Garrit-Jan Boudewijn Van Ommen

Janne Juha Turunen

Annemieke Aartsma-Rus

Petra Van Kuik-Romeijn

Derick Gert Wansink

Peter Adamson

Johannes Antonius Heemskerk

Judith Christina Van Deutekom

RNA editing is achieved using oligonucleotide constructs comprising (i) a targeting portion specific for a target nucleic acid sequence to be edited and (ii) a recruiting portion capable of binding and recruiting a nucleic acid editing entity naturally present in the cell. The nucleic acid editing entity, such as ADAR, is redirected to a preselected target site by means of the targeting portion, thereby promoting editing of preselected nucleotide residues in a region of the target RNA which corresponds to the targeting portion.

Targeted RNA editing

An antisense oligonucleotide capable of preventing or reducing exon 80 inclusion into a human COL7A1 mRNA, and methods for preventing or reducing exon 80 inclusion into a human COL7A1 mRNA.

Antisense oligonucleotides to treat dystrophic epidermolysis bullosa

The invention relates to antisense oligonucleotides (AONs) comprising repetitive trinucleotide units for use in the treatment or prevention of genetic eye diseases, preferably eye dystrophy disorders caused by RNA toxicity such as Fuch's Endothelial Corneal Dystrophy (FECD). The oligonucleotides of the present invention are used to target trinucleotide repeat (TNR) sequence expansions present in intron sequences, to prevent the disease-related sequestration of cellular proteins that interact with such TNR expansions.

Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion

Antisense oligonucleotides to treat Dystrophic Epidermolysis Bullosa

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.

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

The current invention provides for methods and medicaments that apply an oligonucleotide comprising aninosine and/or an uracile and/or a nucleotide containing a base able to form a wobble base pair, said oligonucleotide being preferably RNAse H substantially independent and being complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to a modified oligonucleotide which can be applied in a method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.

Methods and means for treating DNA repeat instability associated genetic disorders

Growing community of inventors

Leiden, Netherlands

Gerardus Johannes Platenburg