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

Venous Thrombosis III: Interprofessional Care01:29

Venous Thrombosis III: Interprofessional Care

Venous thrombosis requires effective prevention and treatment strategies to improve patient outcomes and reduce potential complications.Prevention StrategiesHealthcare providers must prioritize preventing venous thromboembolism (VTE) for all adult patients upon admission. Interventions depend on bleeding and thrombosis risk, medical history, current medications, diagnoses, planned procedures, and patient preferences. Patients on bed rest should change positions every two hours and, if not...
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...

You might also read

Related Articles

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

Sort by
Same author

Adaptive graph learning of microbial phylogeny enables accurate and interpretable microbiome-based host phenotype prediction.

Applied and environmental microbiology·2026
Same author

Cognitive Versus Software-based Fusion Targeted Biopsy for the Diagnosis of Clinically Significant Prostate Cancer: A Multicenter, Randomized, Noninferiority Trial (IMAGINATION).

European urology·2026
Same author

Telerobotic partial nephrectomy and radical prostatectomy using a hybrid network: A single-center prospective experience.

European journal of surgical oncology : the journal of the European Society of Surgical Oncology and the British Association of Surgical Oncology·2026
Same author

Clinical Outcomes of Orchiopexy and the Risk of Malignancy in Postpubertal Cryptorchid Patients.

Andrology·2026
Same author

Size-dependent ruthenium/ceria nanozymes synchronize catalytic ROS scavenging and electrostatic mtDNA sequestration for periodontitis therapy.

Journal of nanobiotechnology·2026
Same author

Bi-A@SR Nanoparticles Enable Tumor-Localized NO Release to Suppress HSP70 and Reverse Chemoresistance.

Advanced healthcare materials·2026

Related Experiment Video

Updated: May 12, 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 Factor Viii for Hemophilia Gene Therapy.

Sean A Roberts1, Biao Dong, Jenni A Firrman

  • 1Sol Sherry Thrombosis Research Center, Philadelphia PA 19140, USA.

Journal of Genetic Syndromes & Gene Therapy
|April 9, 2013
PubMed
Summary

Gene therapy for hemophilia A aims to overcome limitations of factor VIII (fVIII) replacement. Researchers reviewed strategies to engineer fVIII for sustained therapeutic expression, addressing challenges in gene delivery.

More Related Videos

Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes
12:24

Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes

Published on: June 3, 2014

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: May 12, 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

Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes
12:24

Helical Organization of Blood Coagulation Factor VIII on Lipid Nanotubes

Published on: June 3, 2014

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

Area of Science:

  • * Gene therapy
  • * Hematology
  • * Molecular biology

Background:

  • * Current factor VIII (fVIII) replacement therapy for hemophilia A is expensive and risks inhibitor development.
  • * Gene therapy offers a potential alternative to overcome limitations of fVIII replacement.
  • * Hemophilia A gene therapy lags behind hemophilia B due to challenges in expressing the large fVIII protein.

Purpose of the Study:

  • * To review strategies for improving factor VIII (fVIII) gene delivery in hemophilia A.
  • * To discuss the pros and cons of various engineering approaches for fVIII gene therapy.
  • * To address hurdles in achieving long-term, high-level fVIII expression via gene therapy.

Main Methods:

  • * Review of existing literature on engineered factor VIII (fVIII) molecules.
  • * Analysis of viral and non-viral vector strategies for fVIII gene delivery.
  • * Comparative assessment of different approaches to overcome expression challenges.

Main Results:

  • * Various strategies have been developed to engineer the fVIII molecule for enhanced gene delivery.
  • * Challenges remain in achieving sustained therapeutic levels of fVIII expression.
  • * Different engineering approaches present distinct advantages and disadvantages.

Conclusions:

  • * Engineering the fVIII molecule is crucial for advancing hemophilia A gene therapy.
  • * Overcoming delivery and expression hurdles is key to successful fVIII gene therapy.
  • * Further research into optimized fVIII gene strategies is needed for clinical application.