Patents by Inventor WIHELMINA M. C. VAN ROON-MOM

WIHELMINA M. C. VAN ROON-MOM has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES, THE HCHWA-D MUTATION, AND TRINUCLEOTIDE REPEAT EXPANSIONS

Publication number: 20140039037

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.

Type: Application

Filed: October 7, 2013

Publication date: February 6, 2014

Applicant: ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LUMC

Inventors: WIHELMINA M. C. VAN ROON-MOM, MELVIN MAURICE EVERS, BARRY ANTONIUS PEPERS, ANNEMIEKE AARTSMA-RUS, GARRIT-JAN BOUDEWIJN VAN OMMEN

ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES, THE HCHWA-D MUTATION, AND TRINUCLEOTIDE REPEAT EXPANSIONS