Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
2082823 | Drug Discovery Today: Therapeutic Strategies | 2013 | 6 Pages |
RNA targeted therapeutics are being developed in a broad array of therapeutic areas, and more recently a growing number of RNA targeted antisense approaches for cardiovascular and metabolic diseases have progressed into clinical development. Cardiovascular and metabolic diseases are growing health issues with significant associated morbidity and mortality. RNA represents a growing and accessible target space that has been shown to be specifically and selectively targeted utilizing short single strand antisense oligonucleotides. Antisense drugs are relatively small synthetic oligonucleotides with predictable safety and pharmacokinetics across a given chemical platform. Once identified, the remaining risk in development as a therapeutic is associated with selection of the right target, sufficient to affect a clinically meaningful change in the course of disease. This review focuses primarily on the translated mRNAs currently being targeted using antisense therapies in cardiovascular and metabolic disease.