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.
Patent No.:
Date of Patent:
Sep. 01, 2020
Filed:
Oct. 05, 2016
Applicant:
Proqr Therapeutics Ii B.v., Leiden, NL;
Inventors:
Peter Adamson, Leiden, NL;
Janne Juha Turunen, Leiden, NL;
Gerardus Johannes Platenburg, Leiden, NL;
Assignee:
ProQR Therapeutics II B.V., Leiden, NL;
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61P 27/02 (2006.01); A61K 9/00 (2006.01); A61K 31/7105 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/0019 (2013.01); A61K 9/0048 (2013.01); A61K 31/7105 (2013.01); A61P 27/02 (2018.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2320/32 (2013.01);
Abstract
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.