| 摘要 |
<p>A method for directing splicing of a pre-mRNA in a cell capable of performing splicing operation comprising contacting said pre-mRNA in the cell with an agent capable of specifically inhibiting an exon inclusion signal of at least one exon in the pre-mRNA, said method further comprising allowing splicing and translation of the pre-mRNA wherein the translation results in a mutant of normal dystrophin protein and wherein said exon inclusion signal is present in exon number 2, 8, 43, 44, 45, 46, 50, 5, 52 or 53; a method for decreasing the production of aberrant protein in a cell comprising the method of directing splicing; a method for determining whether a nucleic acid comprising complementarity to part of an exon is capable of specifically inhibiting an exon inclusion signal; a nucleic acid capable of specifically inhibiting an exon inclusion signal; a nucleic acid delivery vehicle; and a non-human animal provided with said nucleic acid.</p> |
