Functional validation of allele-specific LMNB1 silencing in patient-derived astrocytes as a therapeutic option for Autosomal Dominant Leukodystrophy
Martina Lorenzati, Arianna Contato, Fernando Josa‐Prado…·Annalisa Buffo
This study validates allele-specific LMNB1 silencing in patient-derived astrocytes as a therapeutic approach for ADLD by showing restored physiological levels and functional improvements.