Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Experimental RNAi02:15

Experimental RNAi

6.2K
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.2K
Recombinant DNA01:09

Recombinant DNA

96.2K
Overview
96.2K
Leaky Scanning02:28

Leaky Scanning

5.2K
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...
5.2K
Types of RNA01:20

Types of RNA

6.1K
Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in regulating gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
RNA Performs Diverse...
6.1K
RACE - Rapid Amplification of cDNA Ends02:35

RACE - Rapid Amplification of cDNA Ends

6.5K
Rapid Amplification of cDNA Ends, or RACE, is one of the most effective methods to obtain a full-length cDNA from an mRNA sequence between a known internal region to the unknown sequence at the 5’ or 3’ end. The unknown region is cloned in the cDNA by a gene-specific primer that binds the known end, and a hybrid primer that attaches a predefined anchor sequence to the unknown end of the cDNA. The sequence in between is amplified by PCR with an anchor primer and a gene-specific...
6.5K
Complementary DNA01:44

Complementary DNA

29.8K
Overview
29.8K

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Corrections to "LAMP2-Mediated Autophagy Plasticity Deters Lysosomal Uptake of Platinum Ions to Reverse Cisplatin-Induced Glioma Progression in a Macrophage Dependent Manner".

Journal of medicinal chemistry·2026
Same author

The Emerging Role of Sialic Acids in Obesity and Diabetes: Molecular Mechanisms and Therapeutic Perspectives.

Biomolecules·2025
Same author

Glycolipids Substitute PEG lipids in Lipid Nanoparticles for mRNA Delivery.

Journal of the American Chemical Society·2025
Same author

mRNA-LNP Vaccines Targeting SmpA-PLD and OmpK-Omp22 Induce Protective Immunity Against <i>Acinetobacter baumannii</i>.

Vaccines·2025
Same author

Development and efficacy of a novel mRNA cocktail for the delivery of African swine fever virus antigens and induction of immune responses.

Microbiology spectrum·2025
Same author

LectoScape: A Highly Multiplexed Imaging Platform for Glycome Analysis and Biomedical Diagnosis.

Analytical chemistry·2024

Related Experiment Video

Updated: Aug 22, 2025

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells
10:07

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells

Published on: November 13, 2014

25.1K

Self-Amplifying RNA Approach for Protein Replacement Therapy.

Dimitri Papukashvili1, Nino Rcheulishvili1, Cong Liu1

  • 1Department of Pharmacology, School of Medicine, Southern University of Science and Technology, Shenzhen 518000, China.

International Journal of Molecular Sciences
|November 11, 2022
PubMed
Summary

Self-amplifying RNA (saRNA) offers advanced protein replacement therapy for various disorders, including rare genetic diseases, with potential for lower doses and fewer side effects than traditional mRNA. Further research is needed for clinical saRNA drug approval.

Keywords:
AATDalpha-1 antitrypsin deficiencymRNAprotein deficiencyprotein replacementsaRNAsingle-gene disorderstaRNA

More Related Videos

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
08:29

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

10.1K
Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells
10:02

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

Published on: June 10, 2022

2.3K

Related Experiment Videos

Last Updated: Aug 22, 2025

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells
10:07

In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells

Published on: November 13, 2014

25.1K
Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
08:29

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

10.1K
Using Lipid Nanoparticles for the Delivery of Chemically Modified mRNA into Mammalian Cells
10:02

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

Published on: June 10, 2022

2.3K

Area of Science:

  • Biotechnology and Genetic Engineering
  • Molecular Biology
  • Therapeutic RNA Technologies

Background:

  • Messenger RNA (mRNA) technology has advanced significantly, with applications beyond infectious disease prevention, including cancer and inherited disorders.
  • The COVID-19 mRNA vaccine approval marks a milestone, opening avenues for broader therapeutic applications of RNA-based approaches.
  • Self-amplifying RNA (saRNA) represents a potentially more effective RNA therapy compared to conventional mRNA.

Purpose of the Study:

  • To review the feasibility of using saRNA for protein replacement therapy across diverse health conditions, including rare hereditary diseases.
  • To provide a comprehensive overview of saRNA technology, encompassing its advantages, molecular structure, mechanism of action, and delivery systems.
  • To highlight the potential of saRNA in overcoming current therapeutic challenges.

Main Methods:

  • Literature review and analysis of existing preclinical and clinical data on saRNA technology.
  • Examination of saRNA's molecular characteristics and biological activity.
  • Assessment of various delivery platforms suitable for saRNA therapeutics.

Main Results:

  • saRNA technology demonstrates potential for protein replacement therapy due to lower dosage requirements, reduced side effects, and sustained therapeutic effects.
  • saRNA exhibits promising characteristics for treating various health disorders, notably rare hereditary diseases.
  • Several delivery platforms are being explored to facilitate effective saRNA-based treatments.

Conclusions:

  • saRNA technology holds significant promise as an advanced therapeutic platform for protein replacement, offering advantages over conventional mRNA.
  • Despite its potential, challenges remain in achieving clinical approval for saRNA-based drugs.
  • Continued research and development in saRNA technology and delivery systems are crucial for its successful translation to clinical practice.