Antisense oligonucleotide therapies in neurodegenerative disease / 国际儿科学杂志

ABSTRACT

Antisense oligonucleotides(ASOs)are small sequences of DNA or RNA able to target mRNA transcripts resulting in change or reduction in the expression of target genes, and became an emerging class of therapeutic drugs.With its high specific, adaptable, and low side effects, ASO drugs are widely used in rare diseases, tumor, inflammatory diseases, cardiovascular disease, and infectious diseases.Recent numerous advancements in ASO sequence design, chemical modifications and delivery methods, significant breakthroughs have been made in the treatment and ASOs become an ideal candidate therapies for neurodegenerative diseases.