Patients with SMA cannot make sufficient amounts of a protein called SMN - short for “survival motorneurone” - which is coded for by a gene called SMN-1. What Suzan Hammond and her colleagues are announcing this week is a DNA sticking plaster or “patch” that can boost the activity of a backup gene, called SMN-2, that can make up for the missing SMN-1.
Susan Hammond is part of the Wood Group.