FIELD: medicine; biotechnology.
SUBSTANCE: present invention relates to the field of biotechnology, specifically to an antisense oligomer conjugate, and can be used in medicine.
EFFECT: invention is directed at constructing a structure that allows to improve the therapeutic index of an antisense oligonucleotide of interest and its release in cells due to the conjugation of oligonucleotide with a cleavable phosphodiester region and a targeting group facilitating the delivery of the resulting construct to the desired location in the cell and providing targeted capture of the active antisense oligomer by the target cells.
21 cl, 25 dwg, 19 tbl, 11 ex
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Authors
Dates
2018-05-08—Published
2013-11-14—Filed