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:
Jan. 14, 2020

Filed:

Mar. 24, 2017
Applicant:

Biomarin Technologies B.v., Leiden, NL;

Inventors:
Assignee:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/04 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3181 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/331 (2013.01); C12N 2310/333 (2013.01); C12N 2310/336 (2013.01); C12N 2320/33 (2013.01);
Abstract

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.


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