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

Gene therapy for hemophilia.

G Hortelano1, P L Chang

  • 1Department of Pathology, McMaster University, Hamilton, Ontario, Canada.

Artificial Cells, Blood Substitutes, and Immobilization Biotechnology
|February 17, 2000
PubMed
Summary

Gene therapy offers a promising alternative for hemophilia A and B by genetically modifying cells to produce clotting factors. Ongoing research aims to overcome challenges for future human clinical trials.

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

Development of the psychometric property of a Minimum Data-Set-Based Depression Rating Scale for use in long-term care facilities in Taiwan.

Aging & mental health·2014
Same author

A de novo transcriptome assembly of Lucilia sericata (Diptera: Calliphoridae) with predicted alternative splices, single nucleotide polymorphisms and transcript expression estimates.

Insect molecular biology·2012
Same author

Encapsulated engineered myoblasts can cure Hurler syndrome: preclinical experiments in the mouse model.

Gene therapy·2011
Same author

Repeated oral administration of chitosan/DNA nanoparticles delivers functional FVIII with the absence of antibodies in hemophilia A mice.

Journal of thrombosis and haemostasis : JTH·2010
Same author

Urinary cell-free DNA as a potential tumor marker for bladder cancer.

The International journal of biological markers·2007
Same author

High inguinal loupe-assisted varicocelectomy for subfertile men with varicococeles: technical feasibility, clinical outcomes and complications.

Archives of andrology·2006

Area of Science:

  • Genetics
  • Molecular Biology
  • Hematology

Background:

  • Hemophilia A and B are X-linked genetic disorders.
  • Current replacement therapy poses health risks and high costs.
  • Gene therapy is being developed as an alternative treatment.

Purpose of the Study:

  • To review current gene therapy approaches for hemophilia.
  • To discuss challenges and progress in the field.

Main Methods:

  • Ex vivo gene therapy: explanting, modifying, and reimplanting patient cells.
  • In vivo gene therapy: direct injection of factor-encoding vectors.
  • Non-autologous gene therapy: using encapsulated engineered cell lines.

Main Results:

  • Various gene therapy strategies are under investigation.
  • Murine and canine models facilitate research.
  • Encouraging progress has been made, despite unresolved issues.

Conclusions:

  • Gene therapy for hemophilia shows significant promise.
  • Further research is needed to address efficacy, safety, and cost.
  • Human clinical trials are anticipated in the near future.

Related Experiment Videos