RNA based antisense drugs: different types, molecular mechanisms and clinical trials

A few decades ago, drugs which were used to treat different diseases were only limited to chemical drugs, peptides, monoclonal antibodies and recombinant proteins. Since the first nucleotide drug was approved by the Food and Drug Organization of the United States, this new generation of medications became remarkable to researchers and pharmacists. Ribonucleic acid (RNA) based drugs were among these category that encountered a number of hurdles along the way such as potential immunogenicity, structural instability and the need for a vehicle to deliver the drug into the cell. With regards to the simplicity of the RNA therapeutics design, researchers could overcome the challenges with improvements in synthetic delivery carriers and chemical modifications of the RNA therapeutics. Numerous types of RNA-based drugs are being extensively tested in various stages of clinical trials. This review focuses on therapeutic ribozymes, antisense oligonucleotides, interfering RNAs and aptamers.