Tiago Outeiro, PhD, University Medical Centre Göttingen, Göttingen, Germany, talks about antisense oligonucleotides (ASOs) and their clinical applications. A more comprehensive understanding of RNA sequencing has enabled the design of ASOs that alter the expression levels and the splicing defects of proteins. Following the approval of ASO therapies for spinal muscular atrophy and Duchenne muscular dystrophy, there has been an immense interest in testing ASOs for other disorders, such as Huntington’s disease and Parkinson’s disease. Although results from recent studies on Huntington’s disease showed disappointing results, future trials on younger patient populations and the use of different doses might alleviate the toxicities observed. Research is also looking into applying ASO therapy to Parkinson’s disease with current strategies targeting the LRRK2 gene and α-synuclein. This interview took place at the 2022 International Congress of Parkinson’s disease and Movement Disorders in Madrid, Spain.
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