Annarita Miccio


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Annarita’s main research interests are the transcriptional control of hematopoiesis, and the development of therapeutic approaches to hematologic genetic disorders. As a PhD student with Giuliana Ferrari at TIGET (Italy), she acquired scientific expertise in hematology and in lentiviral (LV) vector technology for the genetic modification of hematopoietic stem cells (HSCs) and other cell types  (Bonanomi, J Neurosci. 2005; Cattoglio, Blood, 2007). In this context, she generated a β-globin LV and demonstrated rescue of the thalassemic phenotype in a murine model of the disease (Miccio, PNAS, 2008). This LV has been successfully used in an early clinical trial for β-thalassemia and is currently under clinical development. During her postdoctoral period in Gerd Blobel's lab (CHOP, USA) and later as an assistant professor at University of Modena (Italy), she gained experience in epigenetics and the regulation of gene expression in erythroid development and in evaluating the safety and efficacy of gene therapy approaches for hematopoietic disorders (Miccio, EMBO J, 2010; Miccio and Blobel, Mol Cell Biol., 2010 and Gregory, Miccio, Blood, 2010; Moiani, J Clin Invest. 2012). In 2014, Annarita was appointed as a Lab Director at the Imagine Institute (Paris, France), where she pursued her studies on transcriptional regulation in normal and diseased stem cells and their progeny (Cavazza, Stem Cell Reports, 2016; Romano, Scientific Reports, 2016; Antoniani, Stem Cells Transl Med. 2017; Lagresle-Peyrou, Haematologica, 2018). These basic research studies were instrumental in developing novel LV- and genome editing-based strategies for the treatment of β-hemoglobinopathies. In particular, she has optimized the design of a LV expressing an antisickling transgene that is currently employed in a clinical trial for sickle cell disease expected to start in 2019 (Weber, Mol Ther Methods Clin Dev., 2018). In parallel, she developed CRISPR/Cas9 editing strategies for β-hemoglobinopathies (Antoniani, Blood, 2018; Weber, Science Advances, in press) and optimized the delivery of CRISPR/Cas9 in HSPCs (Lattanzi, Mol Ther. 2018).