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Inhibition of MicroRNA-183 Attenuates Pathological TDP-43 Aggregation via SQSTM1/p62 in ALS

Pre-clinical Antisense Oligonucleotide Treatment of CMT2E in a Human Induced Pluripotent Stem Cell (iPSC)-derived Motor Neuron Model

Discovering and Rescuing Toxic Autophagy Derangements in CMT4B3 Using a Novel iPSC-derived Peripheral Nervous System Model of MTMR5 Biology