The infinite possibilities of RNA therapeutics

Abstract

Although the study of RNA therapeutics started decades ago, for many years, this field of research was overshadowed by the growing interest in DNA-based therapies. Nowadays, the role of several types of RNA in cell regulation processes and the development of various diseases have been elucidated, and research in RNA therapeutics is back with force. This short literature review aims to present general aspects of many of the molecules currently used in RNA therapeutics, including in vitro transcribed mRNA, antisense oligonucleotides, aptamers, small interfering RNAs, and microRNAs. In addition, we describe the state of the art of technologies applied for synthetic RNA manufacture and delivery. Likewise, we detail the RNA-based therapies approved by the FDA so far, as well as the ongoing clinical investigations. As a final point, we highlight the current and potential advantages of working on RNA-based therapeutics and how these could lead us to a new era of accessible and personalized healthcare.

Precision polymer nanofibers with a responsive polyelectrolyte corona designed as a modular, functionalizable nanomedicine platform

“…6 One of the most promising applications of polyelectrolytes is the delivery of therapeutic nucleic acids, which remains a key challenge that hampers the development of new treatments for a plethora of diseases from COVID-19 to cancer. 10,11 Numerous barriers exist at both a cellular and organism level that prevent the successful delivery of therapeutics such as nucleic acids to target locations. [12][13][14][15][16] Precision nanomedicine has emerged as a promising solution to overcome many of these barriers, with tailored delivery systems showing particular promise.…”

Length-Controlled Nanofiber Micelleplexes as Efficient Nucleic Acid Delivery Vehicles

“…Nucleic acid (NA) therapeutics show great promise for the treatment of disease and are now recognized as a third major medicine platform alongside traditional small molecules and biologics such as proteins and antibodies . Developments in technologies involving RNA and DNA are leading to new treatments for a plethora of diseases from COVID-19 to cancer. , Despite their promise, the delivery of therapeutic NAs to target locations in vivo remains a key challenge that limits their potential, a task to which lipid nanoparticles and viral vectors are currently the best response. − Several promising applications such as gene therapy and immunotherapy are dependent on the development of effective delivery systems that must overcome a range of issues at organismal, tissue, and cellular levels. − These delivery systems must complex the desired NA cargo in excellent yields, protect it from degradation, and facilitate its release at the target location. − The delivery system must achieve these aims without causing toxicity to the host or eliciting an immune response to the delivery vector. , Precision nanomedicine has emerged as a promising solution to overcome many of these barriers. , To produce precision delivery systems, every aspect of the nanomaterial design must be controlled from the size, shape, and dispersity of the materials to the surface chemistry, rigidity, and localization of functionality.…”

A perspective on oligonucleotide therapy: Approaches to patient customization

“…Because of target specificity, it has become meaningful therapeutics for clinical productivity. The structure and basic sequence of OT directly impact their delivery and potency ( Mollocana-Lara et al, 2021 ).…”

“…Both these oligomers are charge less, resistant to degradation by nucleases, and exhibit variable affinity for the target RNA sequence. ( Mollocana-Lara et al, 2021 ).…”