Related Experiment Video
Updated: Aug 30, 2025
10:07
In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells
Published on: November 13, 2014
25.1K
Optimization of Synthesis of Modified mRNA
1Cardiovascular Research Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Methods in Molecular Biology (Clifton, N.J.)
|August 30, 2022
Summary
Modified mRNA (modRNA) offers a safe, transient gene delivery method without genome integration risks. This study refines modRNA synthesis for enhanced cardiac regeneration therapies.
Area of Science:
- Biotechnology
- Molecular Biology
- Cardiovascular Research
Background:
- Modified mRNA (modRNA) is a safe and effective gene-based therapy vector, as demonstrated by COVID-19 vaccines.
- Traditional gene delivery vectors (plasmids, viruses) face challenges like genome integration and immunogenicity.
- modRNA offers advantages including no genome integration, transient expression, and reduced immunogenicity.
Purpose of the Study:
- To present an updated, optimized, and economical methodology for modRNA synthesis.
- To improve the translational efficiency of modRNA for both in vitro and in vivo applications.
- To highlight the application of modRNA-based therapies in cardiac regeneration.
Main Methods:
- Development and refinement of an optimized modRNA synthesis protocol.
- Evaluation of modRNA translational efficiency in vitro and in vivo.
- Application of modRNA for promoting cardiac regeneration post-myocardial infarction and heart failure.
Main Results:
- The optimized modRNA synthesis method is economical and efficient.
- Enhanced translational efficiency of modRNA was achieved.
- modRNA therapies show potential for cardiac regeneration.
Conclusions:
- modRNA is a superior gene delivery vector compared to traditional methods.
- The refined modRNA synthesis protocol facilitates advanced gene-based therapies.
- modRNA holds significant promise for treating cardiovascular diseases like heart failure.
Related Concept Videos
01:23Regulation of Expression at Multiple Steps
990
The gene expression in cells is regulated at different stages: (i) transcription, (ii) RNA processing, (iii) RNA localization, and (iv) translation. Transcriptional regulation is mediated by regulatory proteins such as transcription factors, activators, or repressors—these control gene expression by initiating or inhibiting the transcription of genes. Once a precursor or pre-mRNA is produced, it undergoes post-transcriptional modification, including 5' capping, splicing, and the...
990
02:51mRNA Stability and Gene Expression
5.7K
The structure and stability of mRNA molecules regulates gene expression, as mRNAs are a key step in the pathway from gene to protein. In eukaryotes, the half-life of mRNA varies from a few minutes up to several days. mRNA stability is essential in growth and development. The absence of the proteins regulating its stability, such as tristetraprolin in mice, can cause systemic issues, including bone marrow overgrowth, inflammation, and autoimmunity.
Cis-acting Elements involved in mRNA stability
Cis-acting Elements involved in mRNA stability
5.7K
01:35Pre-mRNA Processing: Modification of pre-mRNA Ends
9.7K
In eukaryotic cells, transcripts made by RNA polymerase are modified and processed before exiting the nucleus. Unprocessed RNA is called precursor mRNA or pre-mRNA to distinguish it from mature mRNA.
Once about 20-40 ribonucleotides have been joined together by RNA polymerase, a group of enzymes adds a cap to the 5' end of the growing transcript. In this process, a 5' phosphate is replaced by modified guanosine that has a methyl group attached (7-methyl guanosine). This 5' cap helps...
Once about 20-40 ribonucleotides have been joined together by RNA polymerase, a group of enzymes adds a cap to the 5' end of the growing transcript. In this process, a 5' phosphate is replaced by modified guanosine that has a methyl group attached (7-methyl guanosine). This 5' cap helps...
9.7K
01:36What is Gene Expression?
9.0K
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is comprised of nucleotides and proteins are comprised of amino acids, a mediator is required to convert the information encoded in DNA into proteins. This mediator is the messenger RNA (mRNA). mRNA copies the blueprint from DNA by a process called transcription. In eukaryotes, transcription occurs in the nucleus by complementary base-pairing with the DNA template. The mRNA is then...
9.0K
02:24Regulation of Expression Occurs at Multiple Steps
23.0K
Gene expression can be regulated at almost every step from gene to protein. Transcription is the step that is most commonly regulated. This involves the binding of proteins to short regulatory sequences on the DNA. This association can either promote or inhibit the transcription of a gene associated with the respective sequence.
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
Transcription results in the generation of precursor (pre-mRNA) that consists of both exons and introns, which needs further processing before being translated to a...
23.0K
02:31Nuclear Export of mRNA
7.8K
Before mRNAs are exported to the cytoplasm, it is crucial to check each mRNA for structural and functional integrity. Eukaryotic cells use several different mechanisms, collectively known as mRNA surveillance, to look for irregularities in mRNAs. Irregular or aberrant mRNA are rapidly degraded by various enzymes. If a defective mRNA escapes the surveillance, it would be translated into a protein which would either be non-functional or not function properly. One of the primary irregularities in...
7.8K