Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.
- M. Cavazzana‐Calvo, S. Hacein‐Bey, A. Fischer
- Biology, MedicineScience
- 28 April 2000
A gene therapy trial for SCID-X1 was initiated, based on the use of complementary DNA containing a defective gammac Moloney retrovirus-derived vector and ex vivo infection of CD34+ cells, which provided full correction of disease phenotype and clinical benefit.
LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
- S. Hacein-Bey-Abina, C. von Kalle, M. Cavazzana‐Calvo
- Biology, MedicineScience
- 17 October 2003
Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
Artemis, a Novel DNA Double-Strand Break Repair/V(D)J Recombination Protein, Is Mutated in Human Severe Combined Immune Deficiency
- D. Moshous, I. Callebaut, J. Villartay
- BiologyCell
- 20 April 2001
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- N. Cartier, S. Hacein-Bey-Abina, P. Aubourg
- Medicine, BiologyScience
- 6 November 2009
Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- M. Cavazzana‐Calvo, E. Payen, P. Leboulch
- Biology, MedicineNature
- 16 September 2010
It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
- S. Hacein-Bey-Abina, A. Garrigue, M. Cavazzana‐Calvo
- Biology, MedicineJournal of Clinical Investigation
- 2 September 2008
These findings functionally specify a genetic network that controls growth in T cell progenitors and led to sustained remission in 3 of the 4 cases of T cell leukemia, but failed in the fourth.
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.
- S. Hacein-Bey-Abina, C. von Kalle, A. Fischer
- Medicine, BiologyNew England Journal of Medicine
- 16 January 2003
The sustained correction of X-linked severe combined immunodeficiency disease by ex vivo, retrovirally mediated transfer of the γc gene into CD34+ cells in four of five patients with the disease has been reported.
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.
- S. Hacein-Bey-Abina, F. Le Deist, M. Cavazzana‐Calvo
- Medicine, BiologyNew England Journal of Medicine
- 18 April 2002
Ex vivo gene therapy with gamma(c) can safely correct the immune deficiency of patients with X-linked severe combined immunodeficiency and allow patients to have a normal life.
Efficacy of gene therapy for X-linked severe combined immunodeficiency.
- S. Hacein-Bey-Abina, J. Hauer, M. Cavazzana‐Calvo
- Medicine, BiologyNew England Journal of Medicine
- 22 July 2010
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.
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