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RNA-based therapeutics have generated significant attention in recent years due to their potential to treat a variety of chronic and rare diseases, and address targets that have proven to be intractable to antibody and small-molecule approaches. To highlight advances in delivery technologies and improved chemistry that have propelled the clinical advancement of RNA-based therapies (antisense, siRNAs, aptamers, microRNA mimics/anti-miRs and synthetic mRNA), Nature Biotechnology and Nature Reviews Drug Discovery present a joint focus on RNA-based therapies.
RNA-based therapeutics are poised to become successful commercial products, but wide adoption across the biopharmaceutical industry will likely take a few more years.
After years under the radar, RNA vaccines are finally emerging as immunotherapies to combat pandemics and cancer. But as Laura DeFrancesco reports, clinical validation remains elusive.
Non-coding RNAs (ncRNAs) may affect normal gene expression and disease progression, thereby representing potential drug targets. Here, Matsui and Corey assess the potential and challenges in therapeutically exploiting ncRNA species — including microRNA, intronic RNA, repetitive RNA and long ncRNA — highlighting key lessons learned during the development of technologies targeting mRNA.
Refinements in the chemistries employed in oligonucleotide therapeutics have galvanized clinical progress. The complex interplay between chemical modifications and integration into sequence architecture is discussed in the context of antisense and small-interfering RNA drugs.
MicroRNAs (miRNAs) are small non-coding RNAs that can modulate mRNA expression. Insights into the roles of miRNAs in development and disease have led to the development of new therapeutic approaches that are based on miRNA mimics or agents that inhibit their functions (antimiRs), and the first such approaches have entered the clinic. This Review discusses the role of different miRNAs in cancer and other diseases, and provides an overview of current miRNA therapeutics in the clinic.
Nucleic acid aptamers offer several advantages over traditional antibodies, but their clinical translation has been delayed by several factors, including insufficient potency, lack of safety data and high production costs. Here, Zhou and Rossi provide an overview of aptamer generation, focusing on recent technological advances and clinical development, as well as challenges and lessons learned.