Therapeutic levels of fetal hemoglobin in erythroid progeny of  -thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

A zinc-finger transcriptional activator designed to interact with the  -globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts

(2010) A. Wilber et al.   BLOOD

Transcriptional silencing of  -globin by BCL11A involves long-range interactions and cooperation with SOX6

(2010) J. Xu et al.   GENES & DEVELOPMENT

Lentiviral Vector Gene Therapy: Effective and Safe?

(2010) Derek A Persons   MOLECULAR THERAPY

Amelioration of murine  -thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both  -globin and the MGMT drug-resistance gene

(2009) H. Zhao et al.   BLOOD

Ineffective erythropoiesis and thalassemias

(2009) Stefano Rivella   CURRENT OPINION IN HEMATOLOGY

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

 -Thalassemia Treatment Succeeds, With a Caveat

(2009) J. Kaiser   SCIENCE

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY

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

(2008) Phillip W Hargrove et al.   MOLECULAR THERAPY

Artificial MicroRNAs as siRNA Shuttles: Improved Safety as Compared to shRNAs In vitro and In vivo

(2008) Ryan L Boudreau et al.   MOLECULAR THERAPY

Correction of Murine Sickle Cell Disease Using γ-Globin Lentiviral Vectors to Mediate High-level Expression of Fetal Hemoglobin

(2008) Tamara I Pestina et al.   MOLECULAR THERAPY

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

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE

Advances in the allogeneic transplantation for thalassemia

(2007) Guido Lucarelli et al.   BLOOD REVIEWS