Targeted treatment for autosomal dominant leukodystrophy: promising in vitro results

Takeaway

  • Allele-specific silencing by RNA interference (ASP-siRNA) ameliorated protein overexpression and disease-specific phenotypic abnormalities in an in vitro model of autosomal dominant adult-onset demyelinating leukodystrophy (ADLD) due to lamin B overexpression (LMNB1).

Why this matters

  • ASP-siRNA shows promise as a therapeutic option for this fatal condition and could inform development of similar targeted treatments for other pathological conditions linked to gene copy number variations.