HDAC6 Inhibition Corrects Electrophysiological and Axonal Transport Deficits in a Human Stem Cell‐Based Model of Charcot‐Marie‐Tooth Disease (Type 2D) (Adv. Biology 2/2022)

Charcot-Marie-Tooth Disease Treatment In article 2101308, Smith and co-workers use multielectrode arrays to characterize the functional deficits arising in human iPSC-derived spinal neurons bearing Charcot-Marie-Tooth disease type 2D-associated mutations. Loss of activity and network development correlate with reductions in mitochondrial movement, suggesting a defective axonal trafficking mechanism. Observed deficits were ameliorated by treatment with HDAC6 inhibitors, indicating a potential avenue for future therapeutics.