Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

@article{HaceinBeyAbina2002SustainedCO,
  title={Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.},
  author={Salima Hacein-Bey-Abina and Françoise Le Deist and Fr{\'e}d{\'e}rique Carlier and C{\'e}cile Bouneaud and Christophe Hue and Jean-Pierre de Villartay and Adrian J. Thrasher and Nico M. Wulffraat and Ricardo U. Sorensen and Sophie Dupuis-Girod and Alain Fischer and E. Graham Davies and Wietse Kuis and Lilly Leiva and Marina Cavazzana‐Calvo},
  journal={The New England journal of medicine},
  year={2002},
  volume={346 16},
  pages={
          1185-93
        }
}
BACKGROUND X-linked severe combined immunodeficiency due to a mutation in the gene encoding the common gamma (gamma(c)) chain is a lethal condition that can be cured by allogeneic stem-cell transplantation. We investigated whether infusion of autologous hematopoietic stem cells that had been transduced in vitro with the gamma(c) gene can restore the immune system in patients with severe combined immunodeficiency. METHODS CD34+ bone marrow cells from five boys with X-linked severe combined… 

Gene Therapy of X-Linked Severe Combined Immunodeficiency

A successful gene therapy demonstrates that in a setting where transgene expression provides a selective advantage, a clinical benefit can be expected and is used as a basis for a clinical trial of the SCID-X1 disorder caused by common γ (γc) gene mutations.

Correction of canine X-linked severe combined immunodeficiency by in vivo retroviral gene therapy.

This is the first demonstration that in vivo gene therapy targeting HSCs can restore both cellular and humoral immunity in a large-animal model of a fatal immunodeficiency, and achievement of durable immune reconstitution in XSCID dogs is demonstrated.

Efficacy of gene therapy for X-linked severe combined immunodeficiency.

After nearly 10 years of follow-up, gene therapy was shown to have corrected the immunodeficiency associated with SCID-X1 and may be an option for patients who do not have an HLA-identical donor for hematopoietic stem-cell transplantation and for whom the risks are deemed acceptable.

Gene therapy studies in a canine model of X-linked severe combined immunodeficiency.

Gen therapy studies performed in a canine model of XSCID demonstrate that durable T cell reconstitution and thymopoiesis with no evidence of any serious adverse events and sustained marking in myeloid cells and B cells can be achieved for up to 5 years without any pretreatment conditioning.

Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease

This review provides an overview about the different gene therapy approaches used over the last 20 years to treat SCID-X1 patients, particularly focusing on lymphoid immune reconstitution, as well as the developments that have improved the process and outcomes.

Gene Therapy Studies in a Canine Model of X-Linked Severe Combined Immunodeficiency

These studies demonstrate that durable T cell reconstitution and thymopoiesis with no evidence of any serious adverse events and, in contrast to the human XSCID patients, sustained marking in myeloid cells and B cells with Reconstitution of normal humoral immune function can be achieved for up to 5 years without any pretreatment conditioning.

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

The clinical experience of gene therapy for SCID-X1 is put into perspective, with the development and implementation of new generations of safer vectors such as self-inactivating gammaretroviral or lentiviral vectors as well as major advances in integrome knowledge.

[Gene therapy of SCID-X1].

Activation of cellular proto-oncogenes by accidental integration of the gene vector has been identified as the underlying mechanism and improved vector technology in combination with other protocol modifications may reduce the risk of this side effect.
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