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Self-Amplifying RNA Approach for Protein Replacement Therapy.
Papukashvili, Dimitri; Rcheulishvili, Nino; Liu, Cong; Ji, Yang; He, Yunjiao; Wang, Peng George.
Afiliación
  • Papukashvili D; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
  • Rcheulishvili N; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
  • Liu C; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
  • Ji Y; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
  • He Y; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
  • Wang PG; Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.
Int J Mol Sci ; 23(21)2022 Oct 25.
Article en En | MEDLINE | ID: mdl-36361673
Messenger RNA (mRNA) technology has already been successfully tested preclinically and there are ongoing clinical trials for protein replacement purposes; however, more effort has been put into the development of prevention strategies against infectious diseases. Apparently, mRNA vaccine approval against coronavirus disease 2019 (COVID-19) is a landmark for opening new opportunities for managing diverse health disorders based on this approach. Indeed, apart from infectious diseases, it has also been widely tested in numerous directions including cancer prevention and the treatment of inherited disorders. Interestingly, self-amplifying RNA (saRNA)-based technology is believed to display more developed RNA therapy compared with conventional mRNA technique in terms of its lower dosage requirements, relatively fewer side effects, and possessing long-lasting effects. Nevertheless, some challenges still exist that need to be overcome in order to achieve saRNA-based drug approval in clinics. Hence, the current review discusses the feasibility of saRNA utility for protein replacement therapy on various health disorders including rare hereditary diseases and also provides a detailed overview of saRNA advantages, its molecular structure, mechanism of action, and relevant delivery platforms.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: ARN / COVID-19 Límite: Humans Idioma: En Revista: Int J Mol Sci Año: 2022 Tipo del documento: Article País de afiliación: China Pais de publicación: Suiza
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: ARN / COVID-19 Límite: Humans Idioma: En Revista: Int J Mol Sci Año: 2022 Tipo del documento: Article País de afiliación: China Pais de publicación: Suiza