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New antisense oligonucleotide therapies reach first base in ALS

Elia R. Lopez, William F. Borschel & Bryan J. Traynor

January 24, 2022


Two studies highlight the evolution of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for an effective treatment.


In 1993, mutations in the superoxide dismutase 1 (SOD1) gene were the first genetic cause identified in amyotrophic lateral sclerosis (ALS), a neurological disease characterized by rapidly progressive paralysis and death from respiratory failure. Today, an ever-evolving list of genes has been implicated in ALS, also known as Lou Gehrig’s disease after the eponymous baseball player who died of the condition in 1941. Although most cases of ALS are sporadic and of unknown etiology, the cases linked to gene mutations offer an opportunity to use genetic approaches to treat the disease.



Lopez, E. R., Borschel, W. F., & Traynor, B. J. (2022, January 24). New antisense oligonucleotide therapies reach first base in ALS. Nature News. https://www.nature.com/articles/s41591-021-01629-7

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