Advances in the delivery of RNA therapeutics: from concept to clinical reality

The rapid expansion of the available genomic data continues to greatly impact biomedical science and medicine. Fulfilling the clinical potential of genetic discoveries requires the development of therapeutics that can specifically modulate the expression of disease-relevant genes. RNA-based drugs, including short interfering RNAs and antisense oligonucleotides, are particularly promising examples of this newer class of biologics. For over two decades, researchers have been trying to overcome major challenges for utilizing such RNAs in a therapeutic context, including intracellular delivery, stability, and immune response activation. This research is finally beginning to bear fruit as the first RNA drugs gain…

• JD
  Juvenile Diabetes Research Foundation United States of America

  Award: 3-PDF-2017-383-A-N
• NC
  National Cancer Institute

  Awards: P30-CA14051, CA14051, Grant P30-CA14051