About this Research Topic
RNA therapeutics are rapidly coming of age. Already 5 RNA drugs are on the market: one aptamer, two antisense RNAs and two siRNA. They treat macular degeneration, Duchenne muscular dystrophy, spinal muscular atrophy, amyloid cardiomyopathy and hypercholesterolemia. Over 350 clinical trials are in progress.
Regenerative medicine offers an additional area where RNA therapies can have a major impact, using messenger RNA (mRNA) and chemically modified RNA (cmRNA) to express specific regenerative proteins in a strategic fashion, and antisense RNA and RNA interference to suppress gene expression in a highly specific manner. Delivery of RNA has major advantages over traditional gene therapy. It is easier to deliver; the RNA molecules do not need to translocate to the nucleus and the host genome is not disrupted. Although RNA is metabolically much less stable than DNA, this can be an advantage for applications in regenerative medicine where transient expression is not only sufficient, but also desirable. Under present medico-economic conditions, the relative affordability of RNA is also important. Although no RNA therapeutics are yet in clinical trials in the context of regenerative medicine, it is only a matter of time before this happens.
This Research Topic will cover the underlying concepts of RNA therapy as well as recent progress in applying RNA technologies to problems in regenerative medicine. The subject material spans issues concerning the synthesis and production of RNA therapeutics, methods of RNA delivery, the in vitro properties of RNA drugs and their performance in pre-clinical animal models. Production under good manufacturing practice (GMP) conditions and the potential for clinical application will also be considered in the context of the overall RNA therapeutic landscape.
This Research Topic welcomes Original Research, Methods, Mini Reviews, Reviews, Perspective, and Opinion articles covering the following topic areas:
• RNA biology and biochemistry
• RNA modification to improve performance
• RNA delivery using different biomaterials and delivery systems e.g. lipid, polymer, and ceramic nanoparticles
• RNA internalization into cells
• RNA transfer to tissues ex vivo
• RNA transfer to tissues in vivo: preclinical studies on RNA applications for regeneration of different tissues e.g. muscle, cartilage, tendon and bone.
Dr. Christopher Evans is a co-founder of Genascence Inc., and Dr. Elizabeth Balmayor hold patents related to the subject of this Research Topic. All other Topic Editors declare no competing interests with regard to the Research Topic subject.
Keywords: RNA Modifications, RNA Therapeutics, Regenerative Medicine, Non-Viral Gene Therapy, Protein Expression
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