Abstract: 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.
Type:
Grant
Filed:
August 6, 2010
Date of Patent:
September 11, 2012
Assignee:
Prosensa Holding BV
Inventors:
Josephus Johannes de Kimpe, Gerardus Johannes Platenburg
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 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.
Type:
Application
Filed:
August 6, 2010
Publication date:
July 28, 2011
Applicant:
PROSENSA HOLDING BV
Inventors:
Josephus Johannes De Kimpe, Gerardus Johannes Platenburg
Abstract: The use of a sulfurizing agent of formula A: or a salt, hydrate, solvate, or a mixture thereof, in which all R groups, independently, represent H or an organic group, in sulfurization. The sulfurizing agent is preferably a formamidine disulfide. It is found a particularly suitable alternative to existing sulfurizing agents, since it is easy to synthesize from readily available, cheap starting materials.
Type:
Application
Filed:
February 6, 2009
Publication date:
January 20, 2011
Applicant:
PROSENSA HOLDING BV
Inventors:
Peter Christian De Visser, Gerardus Johannes Platenburg