Key result
Gene silencing therapeutics, including antisense oligonucleotides and small interfering RNAs, offer a novel and effective approach to treating cardiovascular diseases such as hyperlipidemias and ATTR amyloidosis.
Gene silencing therapeutics represent a novel and expanding modality in cardiology, allowing for the targeted degradation of mRNA to treat conditions like hypercholesterolemia and cardiac amyloidosis with potentially infrequent dosing.
May expand options for hyperlipidemias and ATTR amyloidosis; leaves open need for RCTs to establish safety and durability.
Therapeutics that inhibit enzymes, receptors, ion channels, and cotransporters have long been the mainstay of cardiovascular medicine. Now, oligonucleotide therapeutics offer a modern variation on this paradigm of protein inhibition. Rather than target a protein, however, small interfering ribonucleic acids and antisense oligonucleotides target the messenger RNA (mRNA) from which a protein is translated. Endogenous, cellular mechanisms enable the oligonucleotides to bind a selected sequence on a target mRNA, leading to its degradation. The catalytic nature of the process [...]
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Jay et al. (2021) conducted a review in Cardiovascular diseases (Hyperlipidemias and Transthyretin-mediated amyloidosis). Gene silencing therapeutics (ASOs and siRNAs) was evaluated. Gene silencing therapeutics, including antisense oligonucleotides and small interfering RNAs, offer a novel and effective approach to treating cardiovascular diseases such as hyperlipidemias and ATTR amyloidosis.
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