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Amyotrophic lateral sclerosis (ALS) is a fatal neurological disease characterized by the degeneration of the corticospinal, medulla, and spinal motor neurons (MN), leading to paralysis and death
FUS protein is an RNA-binding protein, and mutations in FUS are associated with the most aggressive form of early-onset ALS and the rarer form of frontotemporal dementia (FTD)
In FUS mutant mice, the researchers found progressive, age-dependent motor neuron loss due to increased function due to dose-dependent toxicity and associated with insolubility of FUS and related RNA-binding proteins
In ALS patients with FUSP525L mutation, we provide preliminary evidence that repeated intrathecal infusions of ION363 reduce wild-type and mutant FUS levels in the central nervous system, thereby significantly reducing the disease burden of FUS aggregates as a pathological hallmark
In mouse genetic and human clinical studies, the researchers provide evidence supporting FUS silencing as a therapeutic strategy for FUS-dependent ALS and FTD
In mouse genetic and human clinical studies, the researchers provide evidence supporting FUS silencing as a therapeutic strategy for FUS-dependent ALS and FTD
Original source:
Vladislav A.
