FIELD: medicine; biotechnology.
SUBSTANCE: invention refers to molecular biology and medicine, particularly to a short activating nucleic acid molecule for treating MMA. Molecule of a short activating nucleic acid comprises sense and anti-sense nucleic acid chains, which are independent oligonucleotide chains with length of 16 to 35 nucleotides, and one of the nucleotide chains is at least 75 % homologous or complementary to the target nucleotide sequence from the promoter region of the target SMN2 gene. Invention also relates to a pharmaceutical composition containing a short activating nucleic acid molecule, a method of stimulating expression of a target gene in cells and methods of treating a disease caused by insufficient expression of the target gene.
EFFECT: invention provides a novel molecule of a short activating nucleic acid which is capable of activating high-specificity SMN2 transcription by acting on the SMN2 gene promoter.
43 cl, 10 dwg, 9 tbl, 6 ex
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