Gene therapy of lymphoid primary immunodeficiencies
Related Experiment Video
Updated: Oct 10, 2026

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
Published on: May 9, 2025
1Hôpital Necker Enfants Malades, Paris, France. fischer@necker.fr
Abstract:
Gene therapy offers an attractive option to the most severe forms of primary immunodeficiency diseases. Identification of disease-associated genes as well as advances in the technology of gene transfer into hematopoietic progenitor cells have set the basis for the first clinical trials. Settings characterized by the potential for a selective advantage provided to transduced cells are the first diseases to target. The recent example of successful treatment of severe combined immunodeficiency-X1 (gammac deficiency) illustrates this potential.
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