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

25.9K
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...
25.9K
Anticoagulant Drugs: Low-Molecular-Weight Heparins01:30

Anticoagulant Drugs: Low-Molecular-Weight Heparins

919
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...
919
CRISPR01:59

CRISPR

53.0K
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...
53.0K
What is Genetic Engineering?00:49

What is Genetic Engineering?

75.6K
Overview
75.6K
Disorders of Hemostasis01:24

Disorders of Hemostasis

1.2K
Hemostasis, the process that stops bleeding after a blood vessel injury, is crucial for maintaining the integrity of the circulatory system. However, disorders of hemostasis can disrupt this delicate balance, leading to either excessive clotting or bleeding. These disorders can be broadly classified into thromboembolic disorders and bleeding disorders.
Thromboembolic Disorders
Two factors primarily cause thromboembolic conditions.
1.2K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

349
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
349

You might also read

Related Articles

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

Sort by
Same author

Rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP): a Bruton tyrosine kinase inhibitor that can target multiple pathophysiological pathways.

Expert review of hematology·2026
Same author

Efgartigimod as a treatment for adults with primary immune thrombocytopenia: a plain language summary of the ADVANCE IV study.

Therapeutic advances in hematology·2026
Same author

Safety and Effectiveness of Sutimlimab in Cold Agglutinin Disease: A Real-World International Experience.

American journal of hematology·2026
Same author

Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial.

American journal of hematology·2026
Same author

Sutimlimab vs B-cell-targeted therapy in cold agglutinin disease: which is the optimal approach?

Blood·2026
Same author

Warm Autoimmune Haemolytic Anaemia Management in Asia-Pacific: A Delphi Panel Expert Consensus and Systematic Literature Review.

EJHaem·2025

Related Experiment Video

Updated: Sep 16, 2025

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

14.4K

[Gene therapy for hemophilia].

Yoshitaka Miyakawa1

  • 1Department of Hematology, Saitama Medical University.

[Rinsho Ketsueki] the Japanese Journal of Clinical Hematology
|July 6, 2025
PubMed
Summary

Gene therapy for hemophilia A and B offers a new treatment option. Approved in 2022, a single infusion significantly reduces the need for clotting factor injections and bleeding events in most patients.

Area of Science:

  • * Hematology and Gene Therapy
  • * Molecular and Cellular Biology

Background:

  • * Hemophilia A and B are genetic bleeding disorders requiring lifelong clotting factor replacement.
  • * Previous treatments involved frequent intravenous infusions, impacting patients' quality of life.
  • * Gene therapy emerged as a promising alternative to address the root cause of the deficiency.

Purpose of the Study:

  • * To review the recent advancements in gene therapy for hemophilia A and B.
  • * To discuss the clinical trial data and outcomes of approved gene therapies.
  • * To identify the remaining challenges and unmet needs in the field.

Main Methods:

  • * Review of pivotal Phase 3 clinical trials for hemophilia gene therapies.
  • * Analysis of data on efficacy, safety, and durability of treatment outcomes.
Keywords:
AAVGene therapyHemophiliaHemophilia free mind

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

6.5K
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

7.8K

Related Experiment Videos

Last Updated: Sep 16, 2025

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

14.4K
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

6.5K
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

7.8K
  • * Discussion of the vectorology, including adeno-associated virus (AAV) vectors and liver-specific promoters.

    • Main Results:

    • * Gene therapies for hemophilia A and B approved in the US and EU in 2022.
    • * A single adeno-associated virus vector infusion increases clotting factor levels (FVIII/FIX) within weeks.
    • * Approximately 90% of treated patients no longer require factor replacement, with significantly reduced annual bleeding rates.

    Conclusions:

    • * Gene therapy represents a transformative treatment for hemophilia A and B, offering long-term correction.
    • * The success of current therapies highlights the potential of AAV-based gene transfer for genetic disorders.
    • * Further research is needed to address long-term safety, accessibility, and potential for broader application.