Mutation-specific correction of SOD1 in familial ALS using prime and base editing in human induced pluripotent stem cells
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron degeneration and respiratory failure. Approximately 10% of ALS cases are familial, with mutations in SOD1 representing a major genetic cause. Preservation of physiological SOD1 function may be i...
K. Imamura, Kayoko Tsukita, Shin Yoshioka et al.
· BMC Medical Genomics · 0 citations