Introduction to a review series on RNA therapeutics in hematology

Abstract

Small molecule inhibitors and antibody therapies have led the way for targeted therapy of a range of hematologic disorders; however, the number of amenable targets is limited. RNA-directed therapies can be a solution for targets deemed “undruggable,” with modulation of RNA expression through a variety of methods, expanding the therapeutic possibilities. In this Review Series edited by Associate Editor Jason Gotlib, 3 articles highlight areas in which RNA therapeutics are most advanced: acute hepatic porphyria, transthyretin amyloidosis, and hemophilia. This series offers insight into the promise of these new therapies.