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

Gene Therapy00:59

Gene Therapy

25.7K
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...
25.7K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

14.1K
To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
14.1K
CRISPR01:59

CRISPR

52.7K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
52.7K
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

6.1K
Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
6.1K
The Central Dogma01:20

The Central Dogma

23.4K
The central dogma explains the flow of genetic information from DNA nucleotides to the amino acid sequence of proteins.
RNA is the Missing Link Between DNA and Proteins
In the early 1900s, scientists discovered that DNA stores all the information needed for cellular functions and that proteins perform most of these functions. However, the mechanisms of converting genetic information into functional proteins remained unknown for many years. Initially, it was believed that a single gene is...
23.4K
Methods of Nuclear Reprogramming01:24

Methods of Nuclear Reprogramming

1.9K
Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for...
1.9K

You might also read

Related Articles

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

Sort by
Same author

Meeting report: 2025 muscular dystrophy association summit on 'safety and challenges in gene therapy of neuromuscular diseases'.

Journal of neuromuscular diseases·2026
Same author

An Alignment Free Framework for Taxonomic Inference From Codon and Codon-Pair Usage.

Ecology and evolution·2026
Same author

Cell Lines CoCoPUTs: A Database of Codon and Codon-pair Usage Frequencies in Cell Lines.

Journal of molecular biology·2026
Same author

Uncovering position-specific patterns in codon and codon-pair usage in candidate genes associated with blood coagulation diseases.

NAR genomics and bioinformatics·2025
Same author

Editor's Note: MDR1 Synonymous Polymorphisms Alter Transporter Specificity and Protein Stability in a Stable Epithelial Monolayer.

Cancer research·2025
Same author

Uncovering codon usage patterns during murine embryogenesis and tissue-specific developmental diseases.

Frontiers in genetics·2025
Same journal

Targeting developmental reprogramming: hPSC insights for cancer interception.

Trends in pharmacological sciences·2026
Same journal

July 2026 issue first authors.

Trends in pharmacological sciences·2026
Same journal

Chronobiomaterials for circadian-aligned brain therapeutics.

Trends in pharmacological sciences·2026
Same journal

Biosensors for translatable GPCR bias.

Trends in pharmacological sciences·2026
Same journal

ECM stiffness and epigenetics in organ fibrosis.

Trends in pharmacological sciences·2026
Same journal

Which HTT transcript to lower?

Trends in pharmacological sciences·2026
See all related articles

Related Experiment Video

Updated: Aug 23, 2025

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
12:04

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

Published on: March 10, 2023

3.7K

Implementing computational methods in tandem with synonymous gene recoding for therapeutic development.

Brian C Lin1, Nayiri M Kaissarian1, Chava Kimchi-Sarfaty1

  • 1Hemostasis Branch, Division of Plasma Protein Therapeutics, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation & Research, US FDA, Silver Spring, MD, USA.

Trends in Pharmacological Sciences
|October 28, 2022
PubMed
Summary
This summary is machine-generated.

Synonymous gene recoding improves therapeutic development by addressing production limits. Machine learning tools can now assess these recoded therapeutics for safety and efficacy, enhancing variant surveillance.

Keywords:
codon optimizationcodon usagedrug designmachine learningsynonymous variantstherapeutics

More Related Videos

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

6.4K
Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
09:04

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

8.3K

Related Experiment Videos

Last Updated: Aug 23, 2025

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
12:04

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

Published on: March 10, 2023

3.7K
Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

6.4K
Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
09:04

Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells

Published on: September 25, 2019

8.3K

Area of Science:

  • Biotechnology
  • Computational Biology
  • Genetics

Background:

  • Synonymous gene recoding, altering DNA without changing amino acid sequences, is a strategy to overcome therapeutic production challenges.
  • However, evaluating the safety and efficacy of recoded therapeutics is complex due to potential subtle impacts on protein features.
  • Sensitive detection methods are crucial for identifying functional variants introduced by synonymous codon substitutions.

Purpose of the Study:

  • To explore the application of synonymous gene recoding in therapeutic development.
  • To explain the biological mechanisms through which synonymous codon substitutions affect protein features.
  • To investigate the potential of machine-learning (ML) tools for assessing gene-recoded therapeutics and enhancing variant surveillance.

Main Methods:

  • Review of existing literature on synonymous gene recoding in therapeutic development.
  • Explanation of biological mechanisms of synonymous codon substitutions.
  • Exploration of computational approaches, specifically machine learning, for analyzing recoded gene sequences.

Main Results:

  • Synonymous gene recoding has successfully addressed various production limitations in therapeutic development.
  • Synonymous codon substitutions can lead to significant, albeit subtle, changes in protein characteristics.
  • Machine learning tools show promise for evaluating the safety and efficacy of recoded therapeutics.

Conclusions:

  • Synonymous gene recoding is a valuable tool for enhancing therapeutic production.
  • Computational methods, particularly ML, offer powerful capabilities for the surveillance of functional variants in recoded genes.
  • Adapting codon contexts can improve the performance of existing computational tools for therapeutic design.