Annarita Miccio

Annarita Micco completed her Ph.D. with Giuliana Ferrari at TIGET (Italy). She is currently at the Imagine Institute, directing lab of Chromatin and Gene Regulation during Development. Annarita’s main research interests are the transcriptional control of hematopoiesis, and the development of therapeutic approaches to hematologic genetic disorders, and these are reflected in the themes of live projects in her lab, which, have two parallel directions, respectively being the Dynamics of transcriptional and epigenetic networks during stem cell development and Erythropoiesis, and Molecular-based approaches for the treatment of β-Hemoglobinopathies. For the former, her lab has been analyzing genome-wide the occupancy of hematopoietic transcription factors (e.g., GATA1 and GATA2) and their co-factors, and the epigenetic histone modifications associated to transcription or silencing to define regulatory regions involved in hematopoietic stem cell biology and in erythroid commitment and differentiation. The lab utilises these findings for their goal – providing the basic scientific knowledge and and suitable therapeutic targets for sickle cell disease (SCD) and β-thalassemias; they apply established and novel molecular techniques (e.g. genome-wide genomic analyses, lentiviral and CRISPR/Cas9 technologies) by using different cellular models, including clinically relevant hematopoietic stem cells.

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The laboratory of Chromatin and gene regulation during development

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Treatments for β-Hemoglobinopathies 0 Transcriptional and Epigenetic Networks During Stem Cell Development 0 CRISPR/Cas9 0 Lentiviral Vectors 0 Genomic and Bioinformatic Tools 0

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  1. Pavani G, Laurent M, Fabiano A, et al. Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins. Nat Commun. 2020;11(1):4146. Published 2020 Aug 13. doi:10.1038/s41467-020-18036-0

  2. Weber L, Frati G, Felix T, et al. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype. Sci Adv. 2020;6(7):eaay9392. Published 2020 Feb 12. doi:10.1126/sciadv.aay9392

  3. Magnani A, Pondarré C, Bouazza N, et al. Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy. Haematologica. 2020;105(5):1240-1247. doi:10.3324/haematol.2019.227561

  4. Lattanzi A, Meneghini V, Pavani G, et al. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements. Mol Ther. 2019;27(1):137-150. doi:10.1016/j.ymthe.2018.10.008

  5. Magrin E, Miccio A, Cavazzana M. Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies. Blood. 2019;134(15):1203-1213. doi:10.1182/blood.2019000949

  6. Antoniani C, Meneghini V, Lattanzi A, et al. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. Blood. 2018;131(17):1960-1973. doi:10.1182/blood-2017-10-811505


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