The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Date of Patent:
Sep. 11, 2012

Filed:

Aug. 06, 2010
Applicants:

Josephus Johannes DE Kimpe, Utrecht, NL;

Gerardus Johannes Platenburg, Voorschoten, NL;

Inventors:
Assignee:

Prosensa Holding BV, Leiden, NL;

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C07H 21/02 (2006.01); C12N 15/63 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
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.


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