Academisch Ziekenhuis Leiden (h.o.d.n. Lumc)

Academisch Ziekenhuis Leiden

Barry Antonius Pepers

Garrit-Jan Boudewijn Van Ommen

Annemieke Aartsma-Rus

Wilhelmina M C Van Roon-Mom

Melvin Maurice Evers

Wilhelmina Maria Clasina Van Roon-Mom

Wilhelmina Maria Van Roon-Mom

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.

Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions

The invention relates to means and methods for removing a proteolytic cleavage site 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 encodes the proteolytic cleavage site, the method further comprising allowing translation of mRNA produced from the pre-mRNA.

Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins

Described are means and methods for removing a proteolytic cleavage site from a protein, the method comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that encodes the proteolytic cleavage site, and allowing translation of mRNA produced from the pre-mRNA.

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Leiden, Netherlands

Barry Antonius Pepers