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Similarly, a different antisense oligonucleotide linked to BCC10 greatly reduced another gene, Mapt, which encodes the tau protein and is a target for the treatment of Alzheimer's disease and other dementias.

The announcement came a week after drugmakers Biogen and Ionis Pharmaceuticals said they are terminating development of BIIB105, an antisense oligonucleotide for ALS that showed disappointing results in an early-stage clinical study.

The noteworthy aspect of this method is the elimination of the need for specialized and expensive ON building blocks to integrate peptides into the oligonucleotide sequence.

Scientists first imagined creating “antisense oligonucleotide” drugs — pieces of custom-made DNA or RNA designed to correct for genetic errors in cells — in the 1960s.

Another genetic approach that is a bit gray around the categorical edges is known as oligonucleotide therapy.