gene editing

Top 10 SMA stories of 2023

Throughout 2023, SMA News Today brought you the most up-to-date news on scientific breakthroughs and treatment advances related to spinal muscular atrophy (SMA). These are the top 10 most-read articles we published throughout that year, with a brief reminder of what made them relevant to the SMA community. We…

Gene editing of SMN2 leads to normal SMN protein levels: Study

Researchers in the U.S. report using a gene-editing method to alter the SMN2 gene sequence, leading to normal levels of the survival motor neuron (SMN) — the protein that is lacking in spinal muscular atrophy — in early studies. These results were obtained in a cell line derived from…