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

Haemophilia A gene therapy

S Connelly1, M Kaleko

  • 1Genetic Therapy, Inc., Gaithersburg, MD 20878, USA.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|January 5, 1999
PubMed
Summary

Gene therapy offers a promising new treatment for hemophilia A, potentially providing long-term clotting factor expression and improving patient outcomes globally. This approach aims to overcome current treatment limitations and expand access to care worldwide.

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

Investigation of Head Kinematics and Brain Strain Response During Soccer Heading Using a Custom-Fit Instrumented Mouthguard.

Annals of biomedical engineering·2024
Same author

SYN-004 (ribaxamase), an oral beta-lactamase, mitigates antibiotic-mediated dysbiosis in a porcine gut microbiome model.

Journal of applied microbiology·2017
Same author

Direct-acting antiviral drugs for the treatment of chronic hepatitis C virus infection: Interferon free is now.

Clinical pharmacology and therapeutics·2015
Same author

Our experience on Actovegin, is it cutting edge?

International journal of sports medicine·2011
Same author

Natural methods of protein stabilization: thermostable biocatalysts.

Biochemical Society transactions·2007
Same author

Synthesis of adenoviral targeting molecules by intein-mediated protein ligation.

Gene therapy·2003

Area of Science:

  • Hematology
  • Gene Therapy
  • Biotechnology

Background:

  • Current hemophilia A treatment relies on factor VIII (FVIII) replacement therapy.
  • This therapy requires frequent administration and can be inaccessible in remote regions.
  • Gene therapy offers a potential alternative for sustained FVIII expression.

Purpose of the Study:

  • To evaluate the potential of gene therapy for hemophilia A treatment.
  • To highlight recent advancements in gene therapy for hemophilia A.
  • To discuss the feasibility of widespread gene therapy application for hemophilia A.

Main Methods:

  • Development of gene transfer vectors for FVIII expression.
  • Preclinical studies in animal models (mice, dogs, monkeys).
  • Assessment of clotting factor expression levels and phenotypic correction.

Main Results:

  • High-level FVIII expression achieved in preclinical models.
  • Successful phenotypic correction of hemophilia in animal studies.
  • Demonstrated progress in vector technology and sustained expression.

Conclusions:

  • Gene therapy for hemophilia A shows significant promise for improved treatment.
  • Potential for simple, infrequent vector administration could expand global access to care.
  • Ongoing research in vector improvement and sustained expression will make gene therapy a reality.

Related Experiment Videos