Emerging clinical applications of RNA.
RNA is a versatile biological macromolecule that is crucial in mobilizing and interpreting our genetic information. It is not surprising then that researchers have sought to exploit the inherent properties of RNAs so as to interfere with or repair dysfunctional nucleic acids or proteins and to stimulate the production of therapeutic gene products in a variety of pathological situations. The first generation of the resulting RNA therapeutics are now being evaluated in clinical trials, raising significant interest in this emerging area of medical research.
Sullenger
Bruce A
BA
Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA. b.sullenger@cgct.duke.edu
Gilboa
Eli
E
D002986:Clinical Trials as Topic; D003713:Dendritic Cells / Q000378:metabolism / Q000637:transplantation; D005786:Gene Expression Regulation; D005826:Genetics, Medical / Q000379:methods; D006801:Humans; D007167:Immunotherapy / Q000379:methods; D009154:Mutation / Q000235:genetics; D012313:RNA / Q000737:chemistry / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D016337:RNA, Catalytic / Q000737:chemistry / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use; D020040:Trans-Splicing / Q000235:genetics; D014162:Transfection
D016337:RNA, Catalytic; D012313:RNA