abstract |
The present invention establishes molecular therapy for Abel syndrome. The invention relates to an oligonucleotide, a pharmaceutically acceptable salt or a solvate thereof, the oligonucleotide comprising a nucleotide sequence having a nucleotide sequence complementary to the COL4A5 gene of 15-30, And it can induce jumps in exons with truncated mutations and multiples of 3 in the COL4A5 gene of patients with Abel syndrome. The medicine (medicine for treating Abel's syndrome) of the present invention includes the aforementioned oligonucleotide, a pharmaceutically acceptable salt or a solvate thereof. |