University of Delaware researchers have discovered a technique to repair the defective gene that causes spinal muscular atrophy (SMA), the number-one genetic killer of children under two years old in the US.

With the “targeted gene alteration” technique, the research team replaced the function of the SMA-causing defective SMN1 gene by introducing a small fragment of a similar but non-defective gene’s DNA into a diseased cell, triggering the cell to heal itself.

University of Delaware news release