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.
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