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, Marta Ribodino, Maryam Khastkhodaei Ardakani, Ersilia Nicorvo, Elena Signorino, Giacomo Turrini, Valentina Cerrato, Roberta Parolisi, Filippo Lucchini, Gabriele Piacenti, Giacomo Donati, Valentina Proserpio, Juan Antonio García‐León, , 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.
bioRxiv · Sep 15, 2026 · PreprintNeuroscience