Progress in genetic therapies for ALS

Pamela Shaw, DBE, MBBS, MD, FRCP, FMedSci, FAAN, FANA, FAAAS, University of Sheffield, Sheffield, UK, summarizes recent advancements in genetic therapies for the treatment of amyotrophic lateral sclerosis (ALS). A growing number of genetic variants have been associated with ALS, which may account for more than 20% of all ALS cases. Following on from the success of genetic therapies in spinal muscular atrophy (SMA), novel genetic approaches for the treatment of ALS, such as antisense oligonucleotides, have demonstrated significant progress in improving patient outcomes. Prof. Shaw reviews five key therapeutic targets under clinical investigation: superoxide dismutase 1 (SOD1), fused in sarcoma (FUS), chromosome 9 open reading frame 72 (C9orf72), ataxin-2 (ATXN2), and stathmin-2 (STMN2). This interview took place at the 18th Annual Congress on Controversies in Neurology (CONy 2024) in London, UK.
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