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Next-Gen ASO Therapy Targeting CCDC146 to Combat ALS Progression

Novel ASO therapeutic targeting CCDC146, a newly identified driver of motor neuron degeneration in ALS Demonstrated efficacy across both sporadic and familial ALS models, supporting broad patient applicability Improves motor function, neuronal survival, and lifespan in preclinical ALS models Abstract USC researchers have developed a targeted antisense...

2021-224 - Antisense oligonucleotides and CRISPR guide RNAs targeting KCTD20 for the treatment of neurodegeneration

ASOs which target KCTD20 can help reduce neurodegeneration. Selective and targeted treatment, offering durable neuroprotection and clearance of toxic proteins. Can be used to treat neurodegeneration, FTD, ALS with multiple genetic backgrounds. Abstract Glutamate excitotoxicity drives neurodegeneration in ALS and FTD, yet current treatments offer...