| abstract |
A method for at least partially reducing abnormal protein production by a cell, characterized by inducing so-called exon skipping in a cell having an mRNA precursor containing an exon encoding the abnormal protein. Provide a way to do it. Exon skipping results in a mature mRNA that does not have a skipped exon, and if the skipped exon encodes an amino acid, an altered product is obtained. Exon skipping is performed by providing a cell with a reagent that can specifically inhibit an exon inclusion signal of an exon, such as an exon recognition sequence. The exon encapsulation signal can be interfered with by a nucleic acid complementary to a portion of the exon. Also provided are nucleic acids as described above that can be used in the preparation of a medicament, eg, a medicament for the treatment of a genetic disease. [Selection figure] None |