Sleeping Beauty-mediated correction of Fanconi anemia type C

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector

(2010) P S Becker et al.   GENE THERAPY

Cytotoxicity Associated with Artemis Overexpression After Lentiviral Vector-Mediated Gene Transfer

(2010) Megan Multhaup et al.   HUMAN GENE THERAPY

Stable gene transfer and expression in cord blood-derived CD34+ hematopoietic stem and progenitor cells by a hyperactive Sleeping Beauty transposon system

(2009) X. Xue et al.   BLOOD

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

(2009) Lajos Mátés et al.   NATURE GENETICS

Oxidative Stress in Fanconi Anemia Hematopoiesis and Disease Progression

(2008) Wei Du et al.   ANTIOXIDANTS & REDOX SIGNALING