Spinal muscular atrophy (SMA) is caused by a defect in the SMN1 gene, leading to muscle weakness and motor neuron loss. Here, the authors show that combining CRISPR-Cas9 genome editing with gene supplementation improves survival and motor function in SMA mice, highlighting a potential new treatment avenue.
- Fumiyuki Hatanaka
- Keiichiro Suzuki
- Juan Carlos Izpisua Belmonte
