abstract |
An antisense oligonucleotide directed to intron 7 of a nucleic acid molecule encoding SMN2, in which said oligonucleotide has a length of 15, 16, 17, 18, 19 or 20 nucleotides and comprises a 2'-Ometoxyethylazuccan modification in each position, in the that the first nucleotide number (more in 5 ') to which the oligonucleotide seune is nucleotide 122, 123, 124, 125, 126, 127 or 128 of SEQ ID NO: 1. |