FIELD: biotechnology.
SUBSTANCE: invention relates to means and methods of removing a proteolytic cleavage site, a mutation of HCHWA-D or amino acids encoded by an increased number of trinucleotide repeats from a protein, comprising providing a cell which expresses pre-mRNA coding said protein, an antisense oligonucleotide, which induces skipping an exon sequence which contains said proteolytic cleavage site, a HCHWA-D mutation or an increased number of trinucleotide repeats, respectively.
EFFECT: disclosed is antisense oligonucleotide-directed removal of proteolytic cleavage sites, HCHWA-D mutation and increased number of trinucleotide repeats.
6 cl, 20 dwg, 3 tbl, 4 ex
