Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs. Blood. 2020.
Gene therapy on the move. EMBO Mol Med. 2013..
Heterozygous and Homozygous JAK2(V617F) States Modeled by Induced Pluripotent Stem Cells from Myeloproliferative Neoplasm Patients. PLoS One. 2013;8(9):e74257.
Influence of mildly acidic pH conditions on the production of lentiviral and retroviral vectors. Hum Gene Ther Clin Dev. 2014..
Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome. JAMA. 2015;313(15):1550-1563.
Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells. Mol Ther Nucleic Acids. 2013;2:e90..