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

What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
CRISPR01:59

CRISPR

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 Short...
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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.
Gene Therapy00:59

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 inserted. The...
Recombinant DNA01:09

Recombinant DNA

Overview
Homologous Recombination02:31

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

You might also read

Related Articles

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

Sort by
Same author

GH-resistant (Laron) mice: gene therapy with a liver-specific GH receptor causes unbalanced upregulation of female-biased and growth-related genes.

Frontiers in endocrinology·2026
Same author

A protein-based classifier for differentiating follicular thyroid adenoma and carcinoma.

EMBO molecular medicine·2025
Same author

Efficient AAV9 Purification Using a Single-Step AAV9 Magnetic Affinity Beads Isolation.

International journal of molecular sciences·2024
Same author

Transcriptomic convergence despite genomic divergence drive field cancerization in synchronous squamous tumors.

Frontiers in oncology·2024
Same author

Wireless sequential dual light delivery for programmed PDT in vivo.

Light, science & applications·2024
Same author

Author Correction: Artificial intelligence defines protein-based classification of thyroid nodules.

Cell discovery·2022

Related Experiment Video

Updated: Jul 19, 2026

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
09:35

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection

Published on: February 2, 2018

Genetic engineering for haemophilia A.

Shu Uin Gan1, Oi Lian Kon, Roy Y Calne

  • 1National University of Singapore, Department of Surgery, MD11, 04-08, 10 Medical Drive, 117597 Singapore. surgsu@nus.edu.sg

Expert Opinion on Biological Therapy
|September 23, 2006
PubMed
Summary

Gene therapy shows promise for hemophilia A due to a single gene defect. Successful treatment may involve introducing the Factor VIII gene to newborns to induce tolerance and prevent bleeding symptoms.

Area of Science:

  • * Biomedical research
  • * Molecular biology
  • * Hematology

Background:

  • * Hemophilia A is a genetic disorder caused by a deficiency in Factor VIII.
  • * A single gene defect underlies hemophilia A, making it a potential candidate for gene therapy.
  • * Maintaining just 5% of normal Factor VIII levels can prevent severe bleeding.

Purpose of the Study:

  • * To review the current status of gene therapy for hemophilia A.
  • * To outline the challenges and ongoing efforts in developing effective gene therapies.
  • * To discuss potential strategies for overcoming existing obstacles in clinical application.

Main Methods:

  • * Review of existing literature on gene therapy approaches for hemophilia A.
  • * Analysis of experimental results in animal models.

More Related Videos

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

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
08:13

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice

Published on: September 30, 2021

Related Experiment Videos

Last Updated: Jul 19, 2026

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
09:35

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection

Published on: February 2, 2018

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

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice
08:13

Tail Vein Transection Bleeding Model in Fully Anesthetized Hemophilia A Mice

Published on: September 30, 2021

  • * Identification of key challenges and future research directions.

    • Main Results:

    • * Gene therapy for hemophilia A has shown promising results in animal studies.
    • * Introducing the Factor VIII gene to newborn animals before antibody production appears effective.
    • * Inducing a state of tolerance in young animals may be crucial for successful gene therapy.

    Conclusions:

    • * Gene therapy holds significant potential for treating hemophilia A.
    • * Further research and development are necessary before clinical application.
    • * Strategies focusing on early intervention and immune tolerance are key to future success.