FIELD: biotechnology.
SUBSTANCE: disclosed is an antisense oligomer or a pharmaceutically acceptable salt or hydrate thereof, wherein the antisense oligomer induces exon 44 skipping of the human dystrophin gene. Also disclosed is a pharmaceutical composition containing said oligomer or a pharmaceutically acceptable salt or hydrate thereof. Invention also relates to a method of treating muscular dystrophy, which involves administering said oligomer or said pharmaceutical composition to a patient with muscular dystrophy.
EFFECT: invention can be used to produce a drug providing high-efficiency exon skipping.
17 cl, 32 dwg, 7 tbl, 4 ex
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