mRNA Medicine: what’s next after the COVID-19 vaccine? mRNA medicine RNA medicalscience
By Antonia Du Bois, MScReviewed by Danielle Ellis, B.Sc. What is mRNA, and how can it be used therapeutically?Gene editing The coronavirus 2019 pandemic has brought cutting-edge scientific approaches like messenger ribonucleic acid therapeutic technologies into the public eye. In addition to the breakthrough vaccines protecting against severe acute respiratory syndrome coronavirus 2 , mRNA technologies possess a massive potential for gene therapy, anticancer therapy, and regenerative medicine.
While both nucleic acid-based approaches, DNA-based therapies have the drawback of requiring rapid entry into the cytoplasm and nucleus of the cell to be effective. This necessity for nuclear entry represents a potential drawback. mRNA-based therapies improve upon this by not requiring nuclear entry. Additionally, there is no risk of internal mutagenesis of the genome.
Firstly, nucleic acid therapies are far cheaper to produce than protein-based therapies. Secondly, nucleic acids avoid replicating specific post-translational modifications, which can be very difficult to guarantee in protein production. Finally, certain proteins, such as membrane proteins, are unable to be synthesized in vitro, which limits what may be available to researchers.
Immunotherapies Antibodies eBook Compilation of the top interviews, articles, and news in the last year. Download a free copy The general goal of mRNA-based immunotherapy is to elicit immune responses of a specific nature against a target cell. The SARS-CoV-2 vaccines by Pfizer and Moderna represent a well-known example of mRNA-based immunotherapies in the form of a vaccination.
These approaches improve on previous antibody-based therapeutics, which carried problems of reliance on antibody production, low stability, and aggregation. Current studies go beyond CRISPR-Cas-9 to other systems into related approaches, such as Cas-13, to optimize effects in mammalian cells. Cas-13 has shown promise in SARS-CoV-2 detection and the study of facioscapulohumeral muscular dystrophy. Rashnonejad et al. developed Cas-13b gRNAs capable of improving cell viability and decreasing cell death in vivo.
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