Optimizing Modified mRNA In Vitro Synthesis Protocol for Heart Gene Therapy.
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ABSTRACT:
SUBMITTER: Hadas Y
PROVIDER: S-EPMC6722299 | biostudies-literature | 2019 Sep
REPOSITORIES: biostudies-literature
Publications
Optimizing Modified mRNA <i>In Vitro</i> Synthesis Protocol for Heart Gene Therapy.
Hadas Yoav Y Sultana Nishat N Youssef Elias E Sharkar Mohammad Tofael Kabir MTK Kaur Keerat K Chepurko Elena E Zangi Lior L
Molecular therapy. Methods & clinical development 20190730
Synthetic modified RNA (modRNA) is a novel vector for gene transfer to the heart and other organs. modRNA can mediate strong, transient protein expression with minimal induction of the innate immune response and risk for genome integration. modRNA is already being used in several human clinical trials, and its use in basic and translational science is growing. Due to the complexity of preparing modRNA and the high cost of its reagents, there is a need for an improved, cost-efficient protocol to ...[more]
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