FIELD: biotechnology.
SUBSTANCE: group of inventions including a composition of oligonucleotides with controlled chirality capable of selectively suppressing transcripts of a target nucleic acid sequence, a pharmaceutical composition for the treatment of Huntington's disease, a method for cleaving nucleic acid, the use of a composition of oligonucleotides with controlled chirality in a method of treating and/or preventing Huntington's disease and a method of obtaining compositions of oligonucleotides with controlled chirality are described. In one embodiment, the controlled chirality composition comprises oligonucleotides with the same base sequence and length, backbone linker group profile, and backbone chiral centre profile.
EFFECT: invention expands the arsenal of means for selective suppression of the transcript of a target nucleic acid sequence.
59 cl, 41 dwg, 5 tbl, 22 ex
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Authors
Dates
2024-11-22—Published
2016-07-22—Filed