Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

Article Abstract:

Two adults who received gene therapy after nonmyeloablative bone marrow conditioning for the treatment of X-linked chronic granulomatous disease (X-CGD) are reported. Large scale retroviral integration site-distribution analysis showed activating insertion of MDS1-EVI1, PRDM16 or SETBP1 that had influenced regulation of long-term hematopoiesis by expanding gene-corrected myelopoiesis three- or four-fold in both individuals.

Care and treatment, Hematopoietic stem cells, Chronic granulomatous disease

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Effective gene therapy with nonintegrating lentiviral vectors

Article Abstract:

Retroviral and lentiviral vector integration into host-cell chromosomes carries with it a finite chance of causing insertional mutagenesis. A key challenge for clinical therapies based on retroviral vectors is to achieve stable transgene expression while minimizing insertional mutagenesis and in vitro studies have shown that integration-deficient lentiviral vectors can mediate stable transduction.

Chromosomes, Mutagenesis, Retrovirus infections

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Clonality analysis after retroviral-mediated gene transfer to CD34(sup)+ cells from the cord blood of ADA-deficient SCID neonates

Article Abstract:

Results indicate that a single pre-thymic stem or progenitor cell can be the source of the adenosine deaminase gene-marked cells for a prolonged period as revealed by linear amplification-mediated polymerase chain reaction. Data indicate that T-cell clones containing integrants show multiple patterns of T-cell receptor gene rearrangement after 8-year period.

Germany, Analysis, Usage, Physiological aspects, T cells, Stem cells, Gene amplification, Genetic transformation, Severe combined immunodeficiency

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