A novel lentiviral vector targets gene transfer into human hematopoietic stem cells in marrow from patients with bone marrow failure syndrome and in vivo in humanized mice

Measles Virus Glycoprotein-Pseudotyped Lentiviral Vector-Mediated Gene Transfer into Quiescent Lymphocytes Requires Binding to both SLAM and CD46 Entry Receptors

(2011) C. Frecha et al.   JOURNAL OF VIROLOGY

Stem Cell Gene Therapy for Fanconi Anemia: Report from the 1st International Fanconi Anemia Gene Therapy Working Group Meeting

(2011) Jakub Tolar et al.   MOLECULAR THERAPY

Impact of interactions of cellular components of the bone marrow microenvironment on hematopoietic stem and progenitor cell function

(2010) B. R. Chitteti et al.   BLOOD

Knockdown of Fanconi anemia genes in human embryonic stem cells reveals early developmental defects in the hematopoietic lineage

(2010) A. Tulpule et al.   BLOOD

Preclinical and clinical progress in hemophilia gene therapy

(2010) Janka Mátrai et al.   CURRENT OPINION IN HEMATOLOGY

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Targeted gene delivery to CD117-expressing cells in vivo with lentiviral vectors co-displaying stem cell factor and a fusogenic molecule

(2009) Steven Froelich et al.   BIOTECHNOLOGY AND BIOENGINEERING

Efficient and stable transduction of resting B lymphocytes and primary chronic lymphocyte leukemia cells using measles virus gp displaying lentiviral vectors

(2009) C. Frecha et al.   BLOOD

Targeted transduction of CD34+ hematopoietic progenitor cells in nonpurified human mobilized peripheral blood mononuclear cells

(2009) Min Liang et al.   JOURNAL OF GENE MEDICINE

Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

(2009) Francesco Marangoni et al.   MOLECULAR THERAPY

Lentiviral-mediated Genetic Correction of Hematopoietic and Mesenchymal Progenitor Cells From Fanconi Anemia Patients

(2009) Ariana Jacome et al.   MOLECULAR THERAPY

Protection of Stem Cell-Derived Lymphocytes in a Primate AIDS Gene Therapy Model after In Vivo Selection

(2009) Grant D. Trobridge et al.   PLoS One

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

(2009) N. Cartier et al.   SCIENCE

In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1

(2008) P. Rio et al.   BLOOD

Strategies for Targeting Lentiviral Vectors

(2008) Cecilia Frecha et al.   CURRENT GENE THERAPY

Improved lentiviral vectors for Wiskott–Aldrich syndrome gene therapy mimic endogenous expression profiles throughout haematopoiesis

(2008) C Frecha et al.   GENE THERAPY

Deconstructing stem cell self-renewal: genetic insights into cell-cycle regulation

(2008) Keith W. Orford et al.   NATURE REVIEWS GENETICS

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA