siRNA and the lung: research tool or therapeutic drug?
Oligonucleotide-based therapeutics have been hailed as 'the next great wave of the biotechnology revolution' starting with antisense oligonucleotides (ASOs) nearly 20 years ago to RNA interference (RNAi) currently. Is RNAi just the latest research tool or does it have real potential as a therapeutic drug modality? As a research tool, it is evident that RNAi has revolutionized the biological sciences by allowing selective silencing of messenger RNA (mRNA) expression. With the advent of the postgenomic era, RNAi offers a therapeutic platform on which to identify potential picomolar active drug candidates to any target, including those that are conventionally undruggable. In this review, we will discuss the progress made in developing RNAi therapeutics for the treatment of respiratory diseases.
de Fougerolles
Antonin
A
Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142, United States. tdefougerolles@alnylam.com
Novobrantseva
Tatiana
T
D000818:Animals; D002986:Clinical Trials as Topic; D006801:Humans; D007251:Influenza, Human / Q000188:drug therapy; D014677:Pharmaceutical Vehicles; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use*; D018357:Respiratory Syncytial Virus Infections / Q000188:drug therapy; D012140:Respiratory Tract Diseases / Q000188:drug therapy*
D014677:Pharmaceutical Vehicles; D034741:RNA, Small Interfering