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 primary immune deficiencies.

M Otsu1, T Wada, F Candotti

  • 1Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892-1851, USA.

Current Opinion in Allergy and Clinical Immunology
|April 20, 2002
PubMed
Summary

Gene therapy offers hope for primary immunodeficiency diseases by correcting faulty genes. Even low levels of gene transfer can lead to significant clinical improvements, as seen in a recent trial for X-linked severe combined immunodeficiency.

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

Functional Analysis of PTH1R Variants Found in Primary Failure of Eruption.

Journal of dental research·2020
Same author

The long terminal repeat negative control region is a critical element for insertional oncogenesis after gene transfer into hematopoietic progenitors with Moloney murine leukemia viral vectors.

Gene therapy·2016
Same author

Activated STING in a vascular and pulmonary syndrome.

The New England journal of medicine·2014
Same author

Haploinsufficiency of Sf3b1 leads to compromised stem cell function but not to myelodysplasia.

Leukemia·2014
Same author

Development of IgA nephropathy-like glomerulonephritis associated with Wiskott-Aldrich syndrome protein deficiency.

Clinical immunology (Orlando, Fla.)·2011
Same author

Somatic mosaicism caused by monoallelic reversion of a mutation in T cells of a patient with ADA-SCID and the effects of enzyme replacement therapy on the revertant phenotype.

Scandinavian journal of immunology·2011

Area of Science:

  • Immunology
  • Genetics
  • Gene Therapy

Background:

  • Primary immunodeficiency diseases (PIDs) are genetic disorders affecting the immune system.
  • Gene therapy has been explored for PIDs since the 1980s.
  • The potential for gene-corrected cells to outcompete mutated cells offers a unique therapeutic advantage.

Purpose of the Study:

  • To review the progress and potential of gene transfer for primary immunodeficiency diseases.
  • To highlight the significance of selective survival advantage in gene therapy for PIDs.
  • To discuss the recent success in a clinical trial for X-linked severe combined immunodeficiency.

Main Methods:

  • Review of historical gene therapy research in PIDs.
  • Analysis of the biological rationale for gene therapy in PIDs.

Related Experiment Videos

  • Case study of a successful clinical trial for X-linked severe combined immunodeficiency.

    • Main Results:

    • Gene therapy has shown promise for treating primary immunodeficiency diseases.
    • A selective survival advantage of gene-corrected cells can lead to clinical benefits.
    • A recent clinical trial for X-linked severe combined immunodeficiency demonstrated successful outcomes.

    Conclusions:

    • Gene therapy is a viable and evolving approach for primary immunodeficiency diseases.
    • The unique properties of PIDs make them amenable to gene transfer strategies.
    • Continued research and clinical trials are crucial for advancing gene therapy for these conditions.