Antisense oligonucleotides and their applications in rare neurological diseases

Abstract excerpt

Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments for those with rare diseases, more rapid drug discovery strategies are needed. Therapeutic antisense oligonucleotides can modulate target gene expression with...

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• Neurogenetic and Muscular Disorders Research
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• RNA Research and Splicing