title: Synthesis of Modified mRNA for Myocardial Delivery.
abstract: Cardiac gene therapy shows tremendous promise in combating the growing problem of heart disease. Modified mRNA (modRNA) is a novel gene delivery system used in vitro or in vivo to achieve transient expression of therapeutic proteins in a heterogeneous population of cells. Incorporation of specific modified nucleosides enables modRNA to be translated efficiently without triggering antiviral and innate immune responses. ModRNA has been shown to be effective at delivering short-term robust gene expression to the heart and its use in the field of cardiac gene therapy is expanding. Here, we describe a stepwise protocol for the synthesis of modRNA for in vivo myocardial delivery.
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.|Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.|Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
authors.forename: Jason
authors.initials: J
authors.lastname: Kondrat
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA.|Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.|Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
authors.forename: Nishat
authors.initials: N
authors.lastname: Sultana
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Lior.zangi@mssm.edu.|Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Lior.zangi@mssm.edu.|Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Lior.zangi@mssm.edu.
authors.forename: Lior
authors.initials: L
authors.lastname: Zangi
mesh_terms: D000818:Animals; D011232:Chemical Precipitation; D018014:Gene Transfer Techniques; D051379:Mice; D009206:Myocardium / Q000378:metabolism*; D010957:Plasmids / Q000235:genetics; D016133:Polymerase Chain Reaction; D011786:Quality Control; D012333:RNA, Messenger / Q000096:biosynthesis*; D013698:Templates, Genetic; D014158:Transcription, Genetic
chemical_list: D012333:RNA, Messenger
keywords: Gene therapy; In vitro transcription; Modified mRNA; Myocardial delivery; Myocardial infarction