In-vitro Efficacy and In-vivo Biodistribution Studies of an Antisense Oligonucleotide Targeting Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation (S37.010)

This study aimed to explore the efficacy of antisense oligonucleotides (ASOs) to modify DARS2 gene expression in leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL).