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LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

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.
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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

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.
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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

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.
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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.

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

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.
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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.
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Gene Therapy in Patients with Transfusion‐Dependent β‐Thalassemia

Gene therapy with autologous CD34+ cells transduced with the BB305 vector reduced or eliminated the need for long‐term red‐cell transfusions in 22 patients with severe β‐thalassemia without serious adverse events related to the drug product.
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Gene Therapy in a Patient with Sickle Cell Disease: Brief Report

First patient treated with lentiviral vector–mediated addition of an antisickling β‐globin gene into autologous hematopoietic stem cells without recurrence of sickle crises and with correction of the biologic hallmarks of the disease is described.
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Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study

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