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:
Mar. 17, 2020

Filed:

Apr. 21, 2017
Applicant:

Academisch Ziekenhuis Leiden H.o.d.n. Lumc, Leiden, NL;

Inventors:
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/11 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
C12N 15/1137 (2013.01); C12N 15/111 (2013.01); C12N 15/113 (2013.01); C12Y 304/19012 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/346 (2013.01); C12N 2320/33 (2013.01);
Abstract

Described are methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion from a protein comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that comprises the proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of mRNA produced from the pre-mRNA.


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