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mRNA in the Context of Protein Replacement Therapy.
Vavilis, Theofanis; Stamoula, Eleni; Ainatzoglou, Alexandra; Sachinidis, Athanasios; Lamprinou, Malamatenia; Dardalas, Ioannis; Vizirianakis, Ioannis S.
  • Vavilis T; Laboratory of Biology and Genetics, School of Medicine, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.
  • Stamoula E; Department of Dentistry, European University Cyprus, Nicosia 2404, Cyprus.
  • Ainatzoglou A; Centre of Systems Biology, Department of Biotechnology, Biomedical Research Foundation of the Academy of Athens, 11527 Athens, Greece.
  • Sachinidis A; Department of Clinical Pharmacology, School of Medicine, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.
  • Lamprinou M; Centre of Systems Biology, Department of Biotechnology, Biomedical Research Foundation of the Academy of Athens, 11527 Athens, Greece.
  • Dardalas I; Department of Clinical Pharmacology, School of Medicine, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece.
  • Vizirianakis IS; 4th Department of Internal Medicine, Hippokration General Hospital, School of Medicine, Aristotle University of Thessaloniki, 54642 Thessaloniki, Greece.
Pharmaceutics ; 15(1)2023 Jan 03.
Article in English | MEDLINE | ID: covidwho-2166791
ABSTRACT
Protein replacement therapy is an umbrella term used for medical treatments that aim to substitute or replenish specific protein deficiencies that result either from the protein being absent or non-functional due to mutations in affected patients. Traditionally, such an approach requires a well characterized but arduous and expensive protein production procedure that employs in vitro expression and translation of the pharmaceutical protein in host cells, followed by extensive purification steps. In the wake of the SARS-CoV-2 pandemic, mRNA-based pharmaceuticals were recruited to achieve rapid in vivo production of antigens, proving that the in vivo translation of exogenously administered mRNA is nowadays a viable therapeutic option. In addition, the urgency of the situation and worldwide demand for mRNA-based medicine has led to an evolution in relevant technologies, such as in vitro transcription and nanolipid carriers. In this review, we present preclinical and clinical applications of mRNA as a tool for protein replacement therapy, alongside with information pertaining to the manufacture of modified mRNA through in vitro transcription, carriers employed for its intracellular delivery and critical quality attributes pertaining to the finished product.
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Full text: Available Collection: International databases Database: MEDLINE Type of study: Prognostic study Language: English Year: 2023 Document Type: Article Affiliation country: Pharmaceutics15010166

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Full text: Available Collection: International databases Database: MEDLINE Type of study: Prognostic study Language: English Year: 2023 Document Type: Article Affiliation country: Pharmaceutics15010166