New SMN-boosting Molecule Shows Promise as Add-on Therapy

New SMN-boosting Molecule Shows Promise as Add-on Therapy

286766

New SMN-boosting Molecule Shows Promise as Add-on Therapy

Targeting a specific region of an intermediate molecule generated from the SMN2 gene increased the levels of functional SMN — the protein lacking in spinal muscular atrophy (SMA) — in cells derived from SMA patients, a study shows. In addition, combining this approach with a splicing modifier similar to Spinraza (nusinersen) resulted in even greater SMN levels than treatment with the splicing modifier alone. These findings highlighted the therapeutic potential of targeting this region, called 5′ untranslated region, especially…

You must be logged in to read/download the full post.