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 Experiment Videos

Congenital bleeding disorders.

Margaret E Rick1, Christopher E Walsh, Nigel S Key

  • 1National Institutes of Health, Bethesda, MD 20892, USA.

Hematology. American Society of Hematology. Education Program
|November 25, 2003
PubMed
Summary
This summary is machine-generated.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results.

Research and practice in thrombosis and haemostasis·2026
Same author

Relationships between bleeding assessment tools (ISTH-BAT, Self-BAT) and quality of life in patients with bleeding symptoms.

Research and practice in thrombosis and haemostasis·2026
Same author

Rapid detection of fibrinolytic activation in postpartum hemorrhage and acute obstetric coagulopathy using a novel assay.

Blood advances·2025
Same author

Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B.

The New England journal of medicine·2025
Same author

When sickle cell trait is not just trait: risk of VTE.

Hematology. American Society of Hematology. Education Program·2025
Same author

Red cell physiologic stress results in lower quality transfusions: a randomized trial in adults with sickle cell disease.

Blood. Red cells & iron·2025
Same journal

Nze C, Flowers CR. Barriers to accessing cellular therapy for patients receiving care in community practices. Hematology Am Soc Hematol Educ Program. 2023;2023(1):382-385.

Hematology. American Society of Hematology. Education Program·2025
Same journal

CAR T-cell therapy and bispecific antibodies in the management of multiple myeloma.

Hematology. American Society of Hematology. Education Program·2025
Same journal

Emerging immunotherapy advances for non-Hodgkin lymphomas: engaging T cells in the fight.

Hematology. American Society of Hematology. Education Program·2025
Same journal

Anticoagulants in hematologic malignancies: what is the data?

Hematology. American Society of Hematology. Education Program·2025
Same journal

Diagnosis and management of cold agglutinin disease.

Hematology. American Society of Hematology. Education Program·2025
Same journal

What to know about rare B-cell malignancies in 2025.

Hematology. American Society of Hematology. Education Program·2025
See all related articles

Gene therapy offers new hope for hemophilia patients by correcting genetic defects. Research also addresses inhibitor development and improves diagnosis and management of von Willebrand disease for better patient outcomes.

Area of Science:

  • Hematology
  • Genetics
  • Immunology

Background:

  • Congenital bleeding disorders present ongoing clinical and basic science challenges.
  • Hemophilia A and von Willebrand disease are common inherited bleeding conditions requiring advanced management strategies.

Purpose of the Study:

  • To review current advancements in gene therapy for hemophilia.
  • To explore the mechanisms, risk factors, and treatment of inhibitor development in hemophilia A.
  • To enhance the diagnosis and management of von Willebrand disease, particularly in complex cases.

Main Methods:

  • Review of gene transfer principles, viral/non-viral vectors, and alternative therapeutic approaches for hemophilia.
  • Analysis of immunobiology, risk factors, and treatment strategies for inhibitors in hemophilia A, including immune tolerance induction.

Related Experiment Videos

  • Evaluation of diagnostic assays and treatment guidelines for von Willebrand disease based on classification and genetic mutations.

    • Main Results:

    • Gene therapy shows promise for hemophilia, with ongoing trials and future prospects in RNA repair and gene-modified cell therapies.
    • Understanding of inhibitor development in hemophilia A is advancing, with improved diagnostic and treatment protocols, including successful immune tolerance induction.
    • Diagnostic challenges in von Willebrand disease are being addressed through refined laboratory assays and tailored treatment approaches linked to disease classification.

    Conclusions:

    • Gene therapy and novel approaches are poised to revolutionize hemophilia treatment.
    • Comprehensive strategies for inhibitor management are crucial for hemophilia patients.
    • Accurate diagnosis and personalized management are key to improving outcomes for von Willebrand disease patients.