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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.:

US 11427820 B1

PDFFull Text
Date of Patent:
Aug. 30, 2022

Filed:

Sep. 26, 2019

Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna

Applicants:

Biomarin Technologies B.v., Leiden, NL;

Academisch Ziekenhuis Leiden, Leiden, NL;

Inventors:

Josephus Johannes De Kimpe, Utrecht, NL;

Adriana Marie Rus, Hoofddorp, NL;

Gerard Johannes Platenburg, Voorschoten, NL;

Judith Christina Theodora Van Deutekom, Dordrecht, NL;

Garrit-Jan Boudewijn Van Ommen, Amsterdam, NL;

Assignees:

BioMarin Technologies B.V., Leiden, NL;

Academisch Ziekenbuls Leiden, Leiden, NL;

Attorneys:

Rimon, P.C.

Dale L. Rieger

Primary Examiner:

Dana H Shin

Int. Cl.
CPC ...
C12N 15/11 (2006.01); C12N 15/113 (2010.01); A61K 31/56 (2006.01); A61K 31/57 (2006.01); A61K 38/17 (2006.01); A61K 31/7088 (2006.01); A61K 45/06 (2006.01); A61K 31/573 (2006.01); A61K 31/58 (2006.01); A61K 48/00 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 31/56 (2013.01); A61K 31/57 (2013.01); A61K 31/573 (2013.01); A61K 31/58 (2013.01); A61K 31/7088 (2013.01); A61K 38/1719 (2013.01); A61K 45/06 (2013.01); A61K 48/00 (2013.01); C12N 2310/11 (2013.01); C12N 2310/111 (2013.01); C12N 2310/31 (2013.01); C12N 2310/313 (2013.01); C12N 2310/314 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3181 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/346 (2013.01); C12N 2320/31 (2013.01); C12N 2320/33 (2013.01);
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 an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

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