Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

Induced pluripotent stem cells as a tool for gaining new insights into Fanconi anemia

(2012) Lars U.W. Müller et al.   CELL CYCLE

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

Human induced pluripotent stem cells can reach complete terminal maturation: in vivo and in vitro evidence in the erythropoietic differentiation model

(2012) L. Kobari et al.   HAEMATOLOGICA

Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy

(2012) J. E. Adair et al.   Science Translational Medicine

Correction of murine  -thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

(2011) O. Negre et al.   BLOOD

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

(2011) Keshet Ronen et al.   MOLECULAR THERAPY

Analyzing the Number of Common Integration Sites of Viral Vectors – New Methods and Computer Programs

(2011) Ulrich Abel et al.   PLoS One

Forced TR2/TR4 expression in sickle cell disease mice confers enhanced fetal hemoglobin synthesis and alleviated disease phenotypes

(2011) A. D. Campbell et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Estimated Comparative Integration Hotspots Identify Different Behaviors of Retroviral Gene Transfer Vectors

(2011) Alessandro Ambrosi et al.   PLoS Computational Biology

Globin phenotype of erythroid cells derived from human induced pluripotent stem cells

(2010) K.-H. Chang et al.   BLOOD

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

(2010) G. P. Wang et al.   BLOOD

Comparison of human cord blood engraftment between immunocompromised mouse strains

(2010) S. P. McDermott et al.   BLOOD

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

(2010) Eirini P Papapetrou et al.   NATURE BIOTECHNOLOGY

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Cell Cycle and Myc Intersect with Mechanisms that Regulate Pluripotency and Reprogramming

(2009) Amar M. Singh et al.   Cell Stem Cell

Enhanced generation of hematopoietic cells from human hepatocarcinoma cell−stimulated human embryonic and induced pluripotent stem cells

(2009) Min Lu et al.   EXPERIMENTAL HEMATOLOGY

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Efficient Hematopoietic Differentiation of Human Embryonic Stem Cells on Stromal Cells Derived from Hematopoietic Niches

(2008) Maria H. Ledran et al.   Cell Stem Cell

Generation of human-induced pluripotent stem cells

(2008) In-Hyun Park et al.   Nature Protocols