Base editing of SMN2 gene restores production of SMN protein, curing spinal muscular atrophy in mice

A team of medical researchers affiliated with a host of institutions in the U.S. has used base editing to restore the natural production of the SMN protein in mice, effectively curing spinal muscular atrophy (SMA) in the rodents. In their paper published in the journal Science, the group describes their base editing approach and its performance in restoring natural SMN production in mice afflicted with SMA.