Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

(2009) M A Zanta-Boussif et al.   GENE THERAPY

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral Vectors

(2009) Ute Modlich 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

Fanconi anemia and its diagnosis

(2009) Arleen D. Auerbach   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

Unaltered repopulation properties of mouse hematopoietic stem cells transduced with lentiviral vectors

(2008) A. Gonzalez-Murillo et al.   BLOOD

Cancer risks in Fanconi anemia: findings from the German Fanconi Anemia Registry

(2008) P. S. Rosenberg et al.   HAEMATOLOGICA