Related references
Note: Only part of the references are listed.Restoration of NET formation by gene therapy in CGD controls aspergillosis
Matteo Bianchi et al.
BLOOD (2009)
Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.
Alessandro Aiuti et al.
NEW ENGLAND JOURNAL OF MEDICINE (2009)
An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation
Byoung Y. Ryu et al.
BLOOD (2008)
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR)
Schmidt Manfred et al.
NATURE METHODS (2007)
Simian immunodeficiency virus lentivector corrects human X-linked chronic granulomatous disease in the NOD/SCID mouse xenograft
N. Naumann et al.
GENE THERAPY (2007)
Suppression of clonal dominance in cultured human lymphoid cells by addition of the cHS4 insulator to a lentiviral vector
Marguerite V. Evans-Galea et al.
MOLECULAR THERAPY (2007)
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning
H. Bobby Gaspar et al.
MOLECULAR THERAPY (2006)
Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy
Yan Shou et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
MG Ott et al.
NATURE MEDICINE (2006)
Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector
CA Kahl et al.
EXPERIMENTAL HEMATOLOGY (2006)
Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model
EM Kang et al.
EXPERIMENTAL HEMATOLOGY (2006)
Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
B Calmels et al.
BLOOD (2005)
Unusual late presentation of X-linked chronic granulomatous disease in an adult female with a somatic mosaic for a novel mutation in CYBB
B Wolach et al.
BLOOD (2005)
Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: No progression to clonal hematopoiesis or leukemia
HP Kiem et al.
MOLECULAR THERAPY (2004)
Biodistribution of the RD114/mammalian type D retrovirus receptor, RDR
BJ Greens et al.
JOURNAL OF GENE MEDICINE (2004)
Gene therapy insertional mutagenesis insights
UP Davé et al.
SCIENCE (2004)
Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells
P Hematti et al.
PLOS BIOLOGY (2004)
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
S Hacein-Bey-Abina et al.
SCIENCE (2003)
Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/β2-microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells
S Brenner et al.
BLOOD (2003)
Long-term high-level reconstitution of NADPH oxidase activity in murine X-linked chronic granulomatous disease using a bicistronic vector expressing gp91phox and a ΔLNGFR cell surface marker
MA Sadat et al.
HUMAN GENE THERAPY (2003)
Cut it out: Managing hepatic abscesses in patients with chronic granulomatous disease
LE Chen et al.
JOURNAL OF PEDIATRIC SURGERY (2003)
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
S Hacein-Bey-Abina et al.
NEW ENGLAND JOURNAL OF MEDICINE (2003)
Third-generation, self-inactivating gp91phox lentivector corrects the oxidase defect in NOD/SCID mouse-repopulating peripheral blood-mobilized CD34+ cells from patients with X-linked chronic granulomatous disease
J Roesler et al.
BLOOD (2002)
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
A Aiuti et al.
SCIENCE (2002)
Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors
NC Josephson et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
S Hacein-Bey-Abina et al.
NEW ENGLAND JOURNAL OF MEDICINE (2002)
Persistent low-level engraftment of rhesus peripheral blood progenitor cells transduced with the Fanconi anemia C gene after conditioning with low-dose irradiation
EM Kang et al.
MOLECULAR THERAPY (2001)
Chronic granulomatous disease - Report on a national registry of 368 patients
JA Winkelstein et al.
MEDICINE (2000)
Genetic, biochemical, and clinical features of chronic granulomatous disease
BH Segal et al.
MEDICINE (2000)
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
M Cavazzana-Calvo et al.
SCIENCE (2000)