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Review
. 2019 Apr 10;27(4):794-802.
doi: 10.1016/j.ymthe.2018.12.012. Epub 2018 Dec 22.

Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

Zeljka Trepotec  1 , Eva Lichtenegger  2 , Christian Plank  3 , Manish K Aneja  4 , Carsten Rudolph  5
Affiliations
Review

Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

Zeljka Trepotec et al. Mol Ther. .

Abstract

Promising improvements in the field of transcript therapeutics have clearly enhanced the potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are engineered to replace mutated mRNAs and to be immunologically inconspicuous and highly stable while maximizing protein expression. Approaches to deliver mRNA into the cellular cytoplasm safely and efficiently have been further developed so that two mRNA-based approaches replacing vascular endothelial growth factor (VEGF) and cystic fibrosis transmembrane conductance regulator (CFTR) have now made it into clinical trials. These studies bring mRNA therapeutics for protein replacement therapy closer to clinical realization. Herein, we provide an overview of preclinical and clinical developments of mRNA therapeutics for liver diseases.

Keywords: hepatic diseases; mRNA delivery; mRNA therapeutics; protein replacement therapy.

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References

    1. Ramaswamy S., Tonnu N., Tachikawa K., Limphong P., Vega J.B., Karmali P.P., Chivukula P., Verma I.M. Systemic delivery of factor IX messenger RNA for protein replacement therapy. Proc. Natl. Acad. Sci. USA. 2017;114:E1941–E1950. - PMC - PubMed
    1. Kaczmarek J.C., Kowalski P.S., Anderson D.G. Advances in the delivery of RNA therapeutics: from concept to clinical reality. Genome Med. 2017;9:60. - PMC - PubMed
    1. Sahin U., Karikó K., Türeci Ö. mRNA-based therapeutics--developing a new class of drugs. Nat. Rev. Drug Discov. 2014;13:759–780. - PubMed
    1. Dasí F., Benet M., Crespo J., Crespo A., Aliño S.F. Asialofetuin liposome-mediated human α1-antitrypsin gene transfer in vivo results in stationary long-term gene expression. J. Mol. Med. (Berl.) 2001;79:205–212. - PubMed
    1. Brenner S., Jacob F., Meselson M. An unstable intermediate carrying information from genes to ribosomes for protein synthesis. Nature. 1961;190:576–581. - PubMed

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