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Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.

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.

SUBMITTER: Trepotec Z

PROVIDER: S-EPMC6453508 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature

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Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.

Trepotec Zeljka Z Lichtenegger Eva E Plank Christian C Aneja Manish K MK Rudolph Carsten C

Molecular therapy : the journal of the American Society of Gene Therapy 20181222 4

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) ...[more]

PMID: 30655211

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