CNS Specific Antisense Oligonucleotide
Short DNA antisense oligonucleotide constructs having RNA-like regions on both sides of the sequence are known as gapmers. These linear genetic information fragments are designed to hybridise with a target RNA fragment and silence the gene by inducing RNase H cleavage.
However, progress on nucleic acid medications has been gradual, and numerous obstacles must be overcome before their entire impact on patient care can be appreciated. The two most extensively utilised techniques for silencing gene expression are CNS Specific Antisense Oligonucleotide (ASOs) and small interfering RNAs (siRNAs).
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