FIELD: biotechnology.
SUBSTANCE: presents an isolated or purified antisense oligomer for modifying pre-mRNA splicing in a CNOT3 gene transcript, a pharmaceutical composition for treating the effects of a disease associated with CNOT3 expression in a patient, a method of treating a disease associated with CNOT3 expression, and a kit for treating the effects of a disease associated with CNOT3 expression in a patient. Kit contains an antisense oligomer and combinations or cocktails packed in a suitable container together with instructions for use thereof.
EFFECT: invention is aimed at obtaining an antisense oligomer for modifying pre-mRNA splicing in a transcript of the CNOT3 gene or part thereof.
12 cl, 3 tbl, 19 dwg, 6 ex
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