Krizchelle A. Magtoto

LMNB1 reduction is a potential therapeutic strategy in a mouse model of Autosomal Dominant Leukodystrophy

Autosomal dominant leukodystrophy (ADLD) is a fatal-adult-onset CNS demyelinating disorder for which no treatment exists. The majority of ADLD cases are caused by duplications of the lamin B1 ( LMNB1 ) gene, resulting in increased LMNB1 expression. While reducing LMNB1 levels rep...