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

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

Studies in haemoglobin E beta-thalassaemia

(2010) Nancy F. Olivieri et al.   BRITISH JOURNAL OF HAEMATOLOGY

Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy

(2009) B. Cassani et al.   BLOOD

Optimized Lentiviral Vector Design Improves Titer and Transgene Expression of Vectors Containing the Chicken β-Globin Locus HS4 Insulator Element

(2009) Hideki Hanawa et al.   MOLECULAR THERAPY

Genotoxic Potential of Lineage-specific Lentivirus Vectors Carrying the β-Globin Locus Control Region

(2009) Paritha I Arumugam et al.   MOLECULAR THERAPY

Lin28 promotes transformation and is associated with advanced human malignancies

(2009) Srinivas R Viswanathan et al.   NATURE GENETICS

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

(2009) N. Cartier et al.   SCIENCE

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

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Globin Lentiviral Vector Insertions Can Perturb the Expression of Endogenous Genes in β-thalassemic Hematopoietic Cells

(2008) Phillip W Hargrove et al.   MOLECULAR THERAPY

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

(2008) Gary P. Wang et al.   NUCLEIC ACIDS RESEARCH

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