Medicine and HealthNature Communications
Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing
F. Hatanaka, K. Suzuki, et al.
Exciting advancements in spinal muscular atrophy (SMA) treatment have been made by the team led by Fumiyuki Hatanaka and Keiichiro Suzuki. Their innovative CRISPR-Cas9-based strategy offers potential long-term benefits by effectively targeting and correcting SMA mutations in mice, paving the way for promising therapies in inherited diseases.
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