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

Combinatorial Gene Control02:33

Combinatorial Gene Control

Combinatorial gene control is the synergistic action of several transcriptional factors to regulate the expression of a single gene. The absence of one or more of these factors may lead to a significant difference in the level of gene expression or repression.
The expression of more than 30,000 genes is controlled by approximately 2000-3000 transcription factors. This is possible because a single transcription factor can recognize more than one regulatory sequence. The specificity in gene...
General Transcription Factors01:30

General Transcription Factors

Tissue-specific transcription factors contribute to diverse cellular functions in mammals. For example, the gene for beta globin, a major component of hemoglobin, is present in all cells of the body. However, it is only expressed in red blood cells because the transcription factors that can bind to the promoter sequences of the beta globin gene are only expressed in these cells. Tissue-specific transcription factors also ensure that mutations in these factors may impair only the function of...

You might also read

Related Articles

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

Sort by
Same author

Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia models.

Cell reports. Medicine·2026
Same author

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
Same author

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Blood·2026
Same author

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Obstetrical & gynecological survey·2025
Same author

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Obstetrical & gynecological survey·2025
Same author

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.

The New England journal of medicine·2025

Related Experiment Video

Updated: Jun 7, 2026

Overexpressing Long Noncoding RNAs Using Gene-activating CRISPR
13:04

Overexpressing Long Noncoding RNAs Using Gene-activating CRISPR

Published on: March 1, 2019

Codon optimization of human factor VIII cDNAs leads to high-level expression.

Natalie J Ward1, Suzanne M K Buckley, Simon N Waddington

  • 1Molecular Immunology Unit, Institute of Child Health, University College London, London, UK.

Blood
|November 3, 2010
PubMed
Summary

Shorter factor VIII (FVIII) gene therapy constructs improve expression and offer sustained correction in hemophilia A mice. Optimized FVIII gene sequences significantly enhance therapeutic efficacy for hemophilia A treatment.

More Related Videos

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
13:47

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells
10:42

A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells

Published on: December 12, 2017

Related Experiment Videos

Last Updated: Jun 7, 2026

Overexpressing Long Noncoding RNAs Using Gene-activating CRISPR
13:04

Overexpressing Long Noncoding RNAs Using Gene-activating CRISPR

Published on: March 1, 2019

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
13:47

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

Published on: March 29, 2019

A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells
10:42

A Protocol for the Production of Integrase-deficient Lentiviral Vectors for CRISPR/Cas9-mediated Gene Knockout in Dividing Cells

Published on: December 12, 2017

Area of Science:

  • Gene Therapy
  • Hematology
  • Molecular Biology

Background:

  • Hemophilia A gene therapy requires smaller factor VIII (FVIII) expression cassettes for improved efficacy.
  • B domain deleted (BDD) FVIII offers higher expression than wild-type FVIII.
  • Partial BDD FVIII variants, like N6, show enhanced in vitro secretion.

Purpose of the Study:

  • To evaluate smaller, optimized FVIII constructs for enhanced gene therapy in hemophilia A.
  • To assess the in vivo expression and therapeutic efficacy of modified FVIII gene cassettes delivered via lentiviral vectors.

Main Methods:

  • Constructs included various BDD FVIII variants with wild-type or codon-optimized cDNAs.
  • Expression was driven by the Spleen Focus Forming Virus promoter in HIV-based lentiviral vectors.
  • Transduced cells and hemophilic mice were analyzed for FVIII activity and phenotypic correction.

Main Results:

  • Lentiviral vector transduction of 293T cells yielded detectable FVIII activity.
  • Hemophilic mice showed sustained FVIII expression and phenotypic correction for over 250 days.
  • Codon-optimized constructs resulted in a 29- to 44-fold increase in FVIII expression, exceeding 200% of normal levels.

Conclusions:

  • Smaller FVIII constructs are amenable to viral vector delivery and enhance therapeutic efficacy.
  • Optimized FVIII gene sequences significantly boost expression, offering a promising avenue for hemophilia A gene therapy.
  • Further development of these smaller, highly expressed FVIII cassettes could lead to effective gene therapy for hemophilia A.