mRNA therapeutics for musculoskeletal tissue healing


Rosado Balmayor, Elizabeth (RWTH Aachen University Hospital)


"Increasing attention is being paid to the use of RNA molecules as agents to facilitate tissue healing. Messenger RNAs (mRNAs) introduced into the cell induce synthesis of their encoded proteins. This approachholds several advantages over traditional DNA-based gene therapy including improved cell internalization and higher transfection efficiencies, without disruption of the host genome. In the context of tissue engineering and regenerative medicine, the eventual turnover of the exogenous RNA is a further advantage. Because conventional RNAs have strong immunogenicity and low stability, chemical modifications are needed to facilitate their therapeutic application. Moreover, efficient delivery systems are of crucial importance for efficient cell internalization.
This talk will cover progress on mRNA therapeutics with a focus on tissue engineering and regenerative medicine. Particular interest will be placed on applications of mRNA for musculoskeletal regeneration. The presentation will transit from mRNA biology and biochemistry to the internalization of RNA using different biomaterials and non-viral delivery systems, ending with novel applications reported for the regeneration of diverse tissues. In terms of chemical modifications, the use of modified nucleotides, UTRs, and optimized poly(A) tails among other strategies will be reviewed. Regarding RNA delivery, examples of novel lipid and polymer delivery systems will be shown. Also, the advantages of mRNA internalization techniques such as magnetofection will be highlighted. Finally, examples of preclinical studies on mRNA applications to enhance the regeneration of bone tissue will be discussed. Overall, this talk will give an overview of the cmRNA technology from basic aspects to preclinical proof of concepts."


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