rTRD01 is a selective TDP-43 binder that specifically targets the RRM1 and RRM2 domains of TDP-43, partially disrupting its interaction with c9orf72 repeat RNA while preserving binding to canonical sequences. This compound demonstrates significant neuroprotective effects in zebrafish models, improving motor function and providing protection against paraquat-induced neurodegeneration without teratogenic effects at elevated concentrations. rTRD01 is a valuable tool for investigating amyotrophic lateral sclerosis and related TDP-43 proteinopathies, offering insights into the molecular mechanisms involved in these conditions.
rTRD01 is a selective TDP-43 binder that specifically targets the RRM1 and RRM2 domains of TDP-43, partially disrupting its interaction with c9orf72 repeat RNA while preserving binding to canonical sequences. This compound demonstrates significant neuroprotective effects in zebrafish models, improving motor function and providing protection against paraquat-induced neurodegeneration without teratogenic effects at elevated concentrations. rTRD01 is a valuable tool for investigating amyotrophic lateral sclerosis and related TDP-43 proteinopathies, offering insights into the molecular mechanisms involved in these conditions.
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