Mutation-specific correction of SOD1 in familial ALS iPS cells

What was shown

The team compared editing designs for the G93A and L126S variants of SOD1. The best prime-editing setup corrected about 10% of G93A alleles, while base editing corrected about 26% of mutant L126S alleles in patient-derived iPS cells.

Safety: what is and is not known

No above-background edits were found at the predicted off-target sites examined. That does not exclude all off-target risks. Disease-phenotype rescue, delivery, dose and long-term safety remain unproven.

Why it matters

Correcting the causal variant while preserving normal SOD1 function is a rational development strategy. It applies only to a subset of familial ALS and is not currently available to patients.

Related pages

• ALS and iPS research
• Motor-neuron base-editing research
• Understanding conditional approval

Primary sources

• 京都大学iPS細胞研究所（CiRA）、2026-10-09
• Peer-reviewed paper / DOI

Reviewed: 2026-10-12. This page distinguishes laboratory research, clinical trials and approved care. Always verify eligibility and current status with the official institution.