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 hereditary immunodeficiencies]

A Fischer1, G de Saint-Basile, J P Disanto

  • 1INSERM U429, Université René-Descartes, Paris, France.

Comptes Rendus Des Seances De La Societe De Biologie Et De Ses Filiales
|January 1, 1996
PubMed
Summary

Gene therapy for inherited immunodeficiencies faces challenges with current retroviral vectors affecting stem cell integration. However, a selective advantage for transduced cells may enable treatment for conditions like adenosine deaminase deficiency.

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

CD2-induced apoptosis in activated human peripheral T cells: a Fas-independent pathway that requires early protein tyrosine phosphorylation.

Journal of immunology (Baltimore, Md. : 1950)·1996
Same author

gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function.

Blood·1996
Same author

Clinical, hematologic, and biochemical features of a syndrome in Bernese mountain dogs characterized by hepatocerebellar degeneration.

Journal of the American Veterinary Medical Association·1996
Same author

A phase II trial of partially incompatible bone marrow transplantation for high-risk acute lymphoblastic leukaemia in children: prevention of graft rejection with anti-LFA-1 and anti-CD2 antibodies. Société Française de Greffe de Moelle Osseuse.

British journal of haematology·1996
Same author

Radiommunological measurement of leptin in plasma of obese and diabetic human subjects.

Endocrinology·1996
Same author

Basic innervation pattern and distribution of classic autonomic neurotransmitters in human nasal mucosal vasculature.

The Laryngoscope·1996

Area of Science:

  • Immunology
  • Genetics
  • Molecular Biology

Context:

  • Numerous inherited immunodeficiencies involve defects in T cells, B cells, phagocytes, or the complement system.
  • Approximately 20 genes linked to these conditions have been identified, suggesting potential for gene therapy.
  • Current retroviral vectors primarily integrate into cycling cells, posing a challenge for non-cycling hematopoietic stem cells (G0/G1 phase).

Purpose:

  • To explore the potential of gene therapy for severe inherited immunodeficiencies.
  • To address the limitations of current gene transfer vectors for hematopoietic stem cells.
  • To investigate strategies for achieving sustained therapeutic effects in immunodeficient patients.

Summary:

  • Gene therapy for inherited immunodeficiencies is theoretically possible due to identified genes but limited by retroviral vector integration into cycling cells.

Related Experiment Videos

  • Stem cells in the G0/G1 phase are not efficiently targeted by current vectors, hindering widespread application.
  • A selective advantage for transduced stem cells could facilitate lymphocyte differentiation in specific immunodeficiencies with early differentiation blocks, such as adenosine deaminase deficiency (ADA) and X-linked severe combined immunodeficiency (XL SCID).

    • Impact:

    • This research paves the way for clinical studies in ADA-deficient patients.
    • The findings will be evaluated in animal models of XL SCID.
    • Advances in gene therapy could offer definitive cures for severe inherited immunodeficiencies.