SOL-257 Gene Therapy Targeting TDP-43 Shows Promise in Mice

SOL-257 Gene Therapy Targeting TDP-43 Shows Promise in Mice

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SOL-257 Gene Therapy Targeting TDP-43 Shows Promise in Mice

SOL-257, an experimental gene therapy developed by SOLA Biosciences, delayed disease progression and prolonged the lifespan of a mouse model of amyotrophic lateral sclerosis (ALS). The gene therapy is designed to reduce the abnormal TDP-43 protein that forms toxic clumps in the cells of ALS patients. “Targeting only pathogenic [disease-causing] TDP-43 to repair the specific problem has a significant potential for a broadly applicable ALS treatment,” Keizo Koya, PhD, SOLA’s co-founder and CEO, said in…

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