Self-Amplifying RNA Approach for Protein Replacement Therapy

Papukashvili，Dimitri; Rcheulishvili，Nino; Liu，Cong; Ji，Yang; He，Yunjiao; Wang，Peng George

2022-11-01

10.3390/ijms232112884

International Journal of Molecular Sciences   影响因子和分区

1661-6596

1422-0067

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.

AATD alpha-1 antitrypsin deficiency mRNA protein deficiency protein replacement saRNA single-gene disorders taRNA

SCI

英语

第一 ; 通讯

Shenzhen Science and Technology Innovation Program[KQTD20200909113758004];

Biochemistry & Molecular Biology ; Chemistry

Biochemistry & Molecular Biology ; Chemistry, Multidisciplinary

WOS:000882094400001

MDPI

CHEMISTRY

2-s2.0-85141597000

Scopus

Department of Pharmacology,School of Medicine,Southern University of Science and Technology,Shenzhen,518000,China

Papukashvili，Dimitri,Rcheulishvili，Nino,Liu，Cong,et al. Self-Amplifying RNA Approach for Protein Replacement Therapy[J]. International Journal of Molecular Sciences,2022,23(21).

Papukashvili，Dimitri,Rcheulishvili，Nino,Liu，Cong,Ji，Yang,He，Yunjiao,&Wang，Peng George.(2022).Self-Amplifying RNA Approach for Protein Replacement Therapy.International Journal of Molecular Sciences,23(21).

Papukashvili，Dimitri,et al."Self-Amplifying RNA Approach for Protein Replacement Therapy".International Journal of Molecular Sciences 23.21(2022).