Antisense Oligonucleotides Mediated Therapy for Neurodegenerative Disease
Nayak, Apurva
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https://hdl.handle.net/2142/117911
Description
Title
Antisense Oligonucleotides Mediated Therapy for Neurodegenerative Disease
Author(s)
Nayak, Apurva
Issue Date
2022
Keyword(s)
neurodegenerative diseases
Synthetic oligonucleotides
Antisense oligonucleotide-based strategies
Abstract
Many neurodegenerative diseases like Alzheimer’s disease, Huntingtion’s disease, Duchenne’s muscular dystrophy and spinalmuscular 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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