OLIGONUCLEOTIDES ARE COMMONLY MODIFIED AND MEDICINAL USED TO MODULATE THE EXPRESSION OF DUX4

Abstract

The invention relates to a compound and a pharmaceutical intended to normalize the double homeobox 4 (DUX4) of an individual in which the DUX4 gene is abnormally expressed. The invention of the polymorphic oligonucleotide consists of 12 to 30 base pairs. This polymorphic oligonucleotide includes a nucleotide sequence comprising at least 8 contiguous and complementary nucleotide sequences of equal length at positions 126 to 147, 232 to 248, 1306 to 1325, or 1472 to 1495 from the 5' end of the nucleotide of the mature mRNA of DUX4 of SEQ10 NO: 1. The nucleotide sequence of the polymorphic oligonucleotide has at least 90% complementarity with the nucleotide sequence of the mature mRNA of DUX4 of SEQ10 NO: 1.