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

28.3K
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...
28.3K
Gene Therapy00:59

Gene Therapy

4.9K
4.9K
Anticoagulant Drugs: Low-Molecular-Weight Heparins01:30

Anticoagulant Drugs: Low-Molecular-Weight Heparins

2.6K
Hemostasis is a crucial process that prevents excessive blood loss from damaged blood vessels. It involves various mechanisms such as vasoconstriction, platelet adhesion and activation, and fibrin formation. The importance of each mechanism depends on the type of vessel injury. In contrast, thrombosis is the abnormal formation of a blood clot within the blood vessels, leading to potential complications if the clot obstructs blood flow. Thrombosis can be caused by increased coagulability of the...
2.6K
CRISPR01:59

CRISPR

60.4K
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...
60.4K
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

98
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
98
What is Genetic Engineering?00:49

What is Genetic Engineering?

82.0K
Overview
82.0K

You might also read

Related Articles

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

Sort by
Same author

Engineering B cells to express fully customizable antibodies with enhanced Fc functions.

Nature communications·2026
Same author

Engineering immune tolerance for AAV gene therapy: The Norse way.

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

Hemophilia A gene therapy: Moving to the next chapter.

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

Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer.

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

Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.

Journal of blood medicine·2026
Same author

Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.

Blood advances·2026

Related Experiment Video

Updated: Apr 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

15.5K

Gene therapy for hemophilia.

Geoffrey L Rogers1, Roland W Herzog1

  • 1University of Florida, Department of Pediatrics, Division of Cellular and Molecular Therapy, Gainesville, FL 32610.

Frontiers in Bioscience (Landmark Edition)
|January 2, 2015
PubMed
Summary

Gene therapy offers a promising alternative for hemophilia A and B, potentially providing lifelong clotting factor correction with a single treatment. This review explores various gene therapy methods, including viral and nonviral vectors for in vivo and ex vivo applications.

More Related Videos

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

7.9K
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

8.2K

Related Experiment Videos

Last Updated: Apr 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

15.5K
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

7.9K
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

8.2K

Area of Science:

  • Genetics
  • Hematology
  • Molecular Biology

Background:

  • Hemophilia, an X-linked inherited bleeding disorder, presents as hemophilia A or B based on specific mutations.
  • Current treatments involve intravenous clotting factor replacement, which is costly and impacts quality of life.

Purpose of the Study:

  • To review diverse gene therapy strategies for treating hemophilia A and B.
  • To evaluate both in vivo and ex vivo gene therapy approaches.
  • To discuss the use of viral and nonviral delivery vectors in hemophilia gene therapy.

Main Methods:

  • Review of existing literature on gene therapy for hemophilia.
  • Analysis of in vivo and ex vivo treatment modalities.
  • Examination of viral and nonviral vector systems for gene delivery.

Main Results:

  • Gene therapy presents a viable alternative to conventional clotting factor replacement.
  • Various delivery vectors and approaches are under investigation for hemophilia treatment.
  • The goal is to achieve long-term correction of clotting factor activity.

Conclusions:

  • Gene therapy holds significant potential for a one-time, life-long treatment for hemophilia.
  • Further research into efficient and safe delivery systems is crucial.
  • This approach could dramatically improve patient quality of life and reduce healthcare costs.