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Translation

Translation

Lesson: Translation
Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of...

Regulated mRNA Transport

Regulated mRNA Transport

Improving Translational Accuracy

Improving Translational Accuracy

Base complementarity between the three base pairs of mRNA codon and the tRNA anticodon is not a failsafe mechanism. Inaccuracies can range from a single mismatch to no correct base pairing at all. The free energy difference between the correct and nearly correct base pairs can be as small as 3 kcal/ mol. With complementarity being the only proofreading step, the estimated error frequency would be one wrong amino acid in every 100 amino acids incorporated. However, error frequencies observed in...

Leaky Scanning

Leaky Scanning

During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA. Marilyn Kozak discovered that the sequence RCCAUGG (where R...

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Ribosome Profiling

Ribosome Profiling

Ribosome profiling or ribo-sequencing is a deep sequencing technique that produces a snapshot of active translation in a cell. It selectively sequences the mRNAs protected by ribosomes to get an insight into a cell’s translation landscape at any given point in time.
Applications of ribosome profiling
Ribosome profiling has many applications, including in vivo monitoring of translation inside a particular organ or tissue type and quantifying new protein synthesis levels.
The technique...

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Related Experiment Video

Updated: Sep 11, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

Delivering the Message: Translating mRNA Therapy for Liver Inherited Metabolic Diseases.

Sonam Gurung^1,2, Dany Perocheau¹, Roopkatha Ghosh^1,2

¹Great Ormond Street Institute of Child Health, University College London, London, UK.

Journal of Inherited Metabolic Disease

|August 19, 2025

Summary

Messenger RNA (mRNA) lipid nanoparticles (LNPs) offer a revolutionary gene therapy approach. This adaptable mRNA-LNP technology shows promise for treating inherited metabolic diseases, with ongoing clinical trials evaluating its safety and efficacy.

Keywords:

gene therapy inherited metabolic diseases lipid nanoparticle liver mRNA

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Last Updated: Sep 11, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

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Published on: November 4, 2018

Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells

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Published on: June 10, 2022

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Electroporation-Mediated Delivery of Cas9 Ribonucleoproteins and mRNA into Freshly Isolated Primary Mouse Hepatocytes

Published on: June 2, 2022

Area of Science:

Gene Therapy
Nanotechnology
Pharmacology

Background:

Messenger RNA (mRNA) encapsulated in lipid nanoparticles (LNPs) represents a significant advancement in gene therapy.
LNPs serve as non-viral vectors, mitigating immune issues associated with viral gene therapy and offering organ-targeting flexibility.
mRNA-LNP technology has transitioned from vaccines to therapeutics, particularly for inherited metabolic diseases (IMDs).

Purpose of the Study:

To provide an updated overview of mRNA and LNP technologies for treating IMDs.
To review preclinical and clinical studies, focusing on liver-targeted IMDs.
To discuss safety considerations, including infusion reactions and modeling, and future directions for mRNA-LNP therapeutics.

Main Methods:

Review of current mRNA and LNP technologies.
Analysis of preclinical research and ongoing clinical trials for IMDs.
Examination of safety data, including infusion-related reactions and predictive modeling.

Main Results:

mRNA-LNP platforms show promise for IMDs due to liver tropism, with successful preclinical results.
Early-phase clinical trials are underway to assess safety and efficacy in liver IMDs.
The technology's versatility allows for various therapeutic applications, including bridge therapy, long-term cure, and adjuvant treatment.

Conclusions:

mRNA-LNP technology is a rapidly advancing platform with significant potential for treating IMDs.
Translating preclinical success to patient treatment remains a key challenge.
The adaptability of mRNA-LNP therapy positions it as a cornerstone of personalized and individualized gene therapy.