The late-stage results come in advance of pivotal data that Ionis expects to provide for its antisense oligonucleotide ...

A new study shows that lowering MSH3, a key DNA repair protein, with antisense oligonucleotide (ASO) therapy can stop CAG repeat expansions in HD patient-derived brain cells. This could be a promising ...

U of A professor of medical genetics Toshifumi Yokota and the Yokota Lab are making advancements in gene therapy.