FIELD: biotechnology.
SUBSTANCE: group of inventions is described, including a structure for DNA-directed RNA interference (hereinafter – ddRNAi), an expression vector containing the above-mentioned structure, a composition for inhibiting PABPN1 protein expression, which is the cause of oculopharyngeal muscular dystrophy (OPMD), a method for suppressing PABPN1 protein expression, a method for the treatment of oculopharyngeal muscular dystrophy (options) and a kit for the treatment of oculopharyngeal muscular dystrophy. In one of the embodiments of the invention, ddRNAi structure contains DNA sequence encoding hairpin RNA containing effector sequence of at least 17 adjacent nucleotides, which is essentially complementary to RNA transcription area corresponding to PABPN1 protein, where RNA transcription area is given under any of SEQ ID NO: 1-3.
EFFECT: invention expands the arsenal of means for DNA-directed RNA interference.
33 cl, 8 dwg, 5 tbl, 4 ex
| Title | Year | Author | Number |
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