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

26.6K
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...
26.6K
Disorders of Hemostasis01:24

Disorders of Hemostasis

1.6K
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.
1.6K
Blood Transfusion and Agglutination02:45

Blood Transfusion and Agglutination

12.5K
Blood transfusion is a therapeutic measure to restore the blood volume after extensive blood loss due to an accident or a medical procedure. Blood transfusion involves drawing a certain amount of blood from a suitable donor and infusing it into the recipient.
History
The history of blood transfusion dates back to the 17th century, when early attempts were made in animals. In 1818 James Blundell, a British doctor, performed the first successful human blood transfusion. Later in 1900, Karl...
12.5K
Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

634
Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
634
Blood Transfusion01:15

Blood Transfusion

1.3K
Blood transfusion is a critical medical procedure that saves lives and treats various medical conditions. It involves transferring blood from a donor to a recipient. This process requires a thorough understanding of the ABO blood group system and its associated antigens and antibodies.
Blood Transfusion Overview
A blood transfusion is a medical procedure used to replace blood lost due to injury, surgery, or to treat conditions such as anemia or cancer. During a transfusion, donor blood is...
1.3K
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

8.1K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
8.1K

You might also read

Related Articles

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

Sort by
Same author

Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery.

The New England journal of medicine·2026
Same author

Safety, efficacy, and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B.

Blood advances·2026
Same author

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.

Human gene therapy·2026
Same author

Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns.

Molecular therapy : the journal of the American Society of Gene Therapy·2025
Same author

Ancient origin of the furin sequence in the wolf F8 gene.

Open veterinary journal·2025
Same author

"An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B": comment from Lee et al.

Journal of thrombosis and haemostasis : JTH·2025
Same journal

Bridging the Gap in Laboratory Monitoring of Extended Half-Life Factor VIII and IX: Can Thrombin Generation Assays Overcome Assay Discrepancies?

Seminars in thrombosis and hemostasis·2026
Same journal

External Quality Assessment for Low Molecular Weight Heparin Monitoring in the Australasia/Asia-Pacific Region.

Seminars in thrombosis and hemostasis·2026
Same journal

Human evolution: between hemorrhage and thrombosis.

Seminars in thrombosis and hemostasis·2026
Same journal

Full- vs Reduced-Dose Direct Oral Anticoagulants for Extended Treatment of Cancer-Associated Thrombosis: A Multicenter Retrospective Cohort Study.

Seminars in thrombosis and hemostasis·2026
Same journal

Too Old for PESI?: Risk Stratification of Octogenarians with Pulmonary Embolism in the Emergency Department.

Seminars in thrombosis and hemostasis·2026
Same journal

Acute Management and Bleeding Outcomes in Pediatric Pulmonary Embolism: A Large Single-Center Retrospective Cohort Study.

Seminars in thrombosis and hemostasis·2026
See all related articles

Related Experiment Video

Updated: Nov 16, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.0K

Gene Therapy for Inherited Bleeding Disorders.

Valder R Arruda1,2,3, Jesse Weber2, Benjamin J Samelson-Jones1,2,3

  • 1Department of Pediatrics, Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.

Seminars in Thrombosis and Hemostasis
|February 26, 2021
PubMed
Summary
This summary is machine-generated.

Gene therapy for hemophilia is nearing regulatory approval, overcoming significant challenges with advancements in viral vectors and specific gene targets. Lessons learned are guiding development for other rare bleeding disorders.

More Related Videos

Characterization of Blood Outgrowth Endothelial Cells BOEC from Porcine Peripheral Blood
08:02

Characterization of Blood Outgrowth Endothelial Cells BOEC from Porcine Peripheral Blood

Published on: January 6, 2022

1.9K
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

7.9K

Related Experiment Videos

Last Updated: Nov 16, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.0K
Characterization of Blood Outgrowth Endothelial Cells BOEC from Porcine Peripheral Blood
08:02

Characterization of Blood Outgrowth Endothelial Cells BOEC from Porcine Peripheral Blood

Published on: January 6, 2022

1.9K
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

7.9K

Area of Science:

  • * Hematology and Gene Therapy
  • * Molecular and Translational Medicine

Background:

  • * Decades of research in gene therapy for hemophilia A and B.
  • * Gene therapy field advancements, including adeno-associated viral vectors.
  • * Disease-specific breakthroughs like B-domain deleted factor VIII and factor IX Padua.

Purpose of the Study:

  • * Review the progress and challenges in hemophilia gene therapy.
  • * Discuss the current status of gene therapy for other rare inherited bleeding disorders.
  • * Extrapolate lessons from hemophilia to guide future clinical development.

Main Methods:

  • * Review of preclinical and clinical studies in gene therapy for hemophilia.
  • * Analysis of advancements in viral vector technology.
  • * Examination of disease-specific genetic modifications and their impact.

Main Results:

  • * Promising pivotal studies suggest imminent regulatory approval for hemophilia gene therapy.
  • * Hemophilia B studies identified critical safety concerns regarding immune responses to vector capsids.
  • * Preclinical investigations are underway for factor VII deficiency, von Willebrand disease, and Glanzmann thrombasthenia.

Conclusions:

  • * Gene therapy for hemophilia is a significant achievement, built upon overcoming numerous hurdles.
  • * Safety insights from hemophilia B trials are crucial for future gene therapy endeavors.
  • * The trajectory of hemophilia gene therapy offers a roadmap for treating other rare inherited bleeding disorders.