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LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1
TLDR
Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter. Expand
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
TLDR
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. Expand
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
TLDR
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. Expand
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
TLDR
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. Expand
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.
TLDR
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. Expand
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.
TLDR
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. Expand
Efficacy of gene therapy for X-linked severe combined immunodeficiency.
TLDR
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. Expand
Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study
TLDR
The results show that ex-vivo selective depletion of T cells that cause graft-versus-host disease is efficient and feasible, even in haploidentical settings. Expand
Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial.
TLDR
A longitudinal study of gene-corrected progenitor cell populations from 8 patients using 454 pyrosequencing to map vector integration sites, and extensive resampling to allow quantification of clonal abundance, emphasizes that key features of transduced cell populations--including diversity, integration site clustering, and expansion of some clones--were established early after transplantation. Expand
Severe combined immunodeficiency. A model disease for molecular immunology and therapy
TLDR
Based on the assumption that long‐lasting pluripotent progenitor cells are transduced, these data suggest that gene therapy could overcome the long‐term recurrence of the T‐cell immunodeficiency. Expand
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