Rostudirsen

Rostudirsen is an oligonucleotide and a component of Zelecimentrostudirsen. This compound is created by linking Rostudirsen with a fragment antibody known as Zeleciment. Zeleciment specifically binds to transferrin receptor 1 (TfR1), which is highly expressed in muscle tissue. Zelecimentrostudirsen targets muscle tissue, facilitating exon 51 skipping within cells, thus enabling muscle cells to produce dystrophin protein close to its full-length form.