… While this can be centered on the potency of the antisense oligonucleotide itself, the rapid
release/availability of the antisense sequence can also account for this observation, since it …

… Nucleic acid modifications also modulate the response of … D, it is metabolized to a non-toxic
metabolite C. It is important … Together, these studies suggest that inclisiran is a potent PCSK9 …

… Exondys 51 is an antisense oligonucleotide modified by phosphoramidate … The potency in
vivo was potent with a whole 100 mg/kg dosage without a delivery system. Also, as contained …

… and antisense oligonucleotides. … antisense oligonucleotide conjugated with hydrophobic
disulfide modifications is generated for their ability to selfassemble into spherical nucleic acids …

… Nucleic acid-based therapeutic can be further subdivided … are often referred to as antisense
oligonucleotides (ASO). … critical for the potency of the final product, as it is a potent inducer of …

… nucleic acid sequences to the target tissue. Recent developments in biotechnology have
enabled powerful … of biology, which has resulted in nucleic acid-based therapeutics becoming a …

… leads to efficient entrapment of nucleic acid with enhanced in-… In bulk mixing process, the
strong and fast interactions … This leads to the bottom-up synthesis of liposomes, with a strong …

… in the CNS without impairing its potent biological activity. In the … The administration of ASO5-2
was non-toxic in mice as … in this study: green circle, locked nucleic acid (LNA); blue circle, …

… potency and relevance for clinical use. ASOs are short single-stranded sequences of nucleic
acid… nucleus while being non-toxic and non-immunologic for cells. Encapsidation of vectors …

… As such, across both sequences we observed strong sensitivity to a single mismatch in key
regions, while mismatches in other regions maintained full activity. This is consistent with the …