Antisense Oligonucleotides Mediated Therapy for Neurodegenerative Disease

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Apurva Nayak


Many neurodegenerative diseases like Alzheimer’s disease, Huntingtion’s disease, Duchenne’s muscular dystrophy and spinal muscular atrophy are linked to aggregated, toxic proteins. Antisense oligonucleotide-based strategies (ASOs) are the most direct method of targeting gene expression. Synthetic oligonucleotides bind to the target mRNA by Watson-Crick hybridization and can either promote the degradation of RNA or inhibit it. In 2016, two ASO therapies for spinal muscular atrophy and Duchenne muscular dystrophy were approved by the FDA.

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Author Biography

Apurva Nayak

Apurva is a Senior majoring in Molecular and Cellular Biology with minors in Sociology and Chemistry on the pre-med track. She currently works on campus as a Research Assistant in the Physical Activity and Neurocognitive Health Lab and is a student worker at McKinley. She is excited to be a part of this volume of Brain Matters to increase discussion.