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13 - 04 - 2017

Gene extinction to treat ALS and spinocerebellar ataxia

Two studies published yesterday showed that antisense oligonucleotides (molecules that inactivate a gene) could be a therapy for serious diseases today, without treatment.

Antisense nucleotides directed against the ataxin 2 gene have slowed down the onset of spinocerebellar ataxia type 2 in a mouse model, as well as the onset of amyotrophic lateral sclerosis in another mouse model.

These results follow years of research in yeasts and flies. Other preclinical studies are planned with the end game to treat these diseases.