Development of Antisense Gapmers for the Treatment of Huntington's Disease. (PubMed, Methods Mol Biol) - Oct 1, 2026 - "Antisense-mediated knockdown of mutated huntingtin is a promising therapeutic approach for Huntington's disease (HD), a devastating disorder affecting motor and cognitive abilities. This chapter focuses on the modified gapmer AOs for the treatment of HD."
Journal • CNS Disorders • Huntington's Disease • Movement Disorders
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Tejal Aslesh; Toshifumi Yokota
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