Obtained his degree in Chemistry in 2001 and his Ph.D. in Biochemistry in 2005 at the Department of Biochemical Sciences “A. Rossi Fanelli” of the “Sapienza” University of Rome. Here he stayed as Post-doc until 2011. Currently, he is employed as assistant Professor at the Department of Life, Health and Environmental Sciences at the University of L’ Aquila. Francesco Angelucci is mainly involved in the study of structure-function relationships of potential drug-targets from Schistosoma, the pathological agent of schistosomiasis. The final purpose of this approach is to find new therapies against this neglected disease that affects 280 million people in tropical and sub-tropical countries.




Degree in chemistry in 2002 at the University of Rome “La Sapienza” studying the mechanism of oxidation of benzylic alcohols and ethers from a laccase of Trametes versicolor. PhD. in Biochemistry at the Institute A. Rossi Fanelli of the University “La Sapienza” working in the crystallography group of Prof. M. Brunori. Her Ph.D project was focused on the structural and functional study of pathogenically important proteins from Schistosoma mansoni. In 2006, she worked at IRBM in the crystallography group under the guide of Dr. S. Di Marco. Her study on the allosteric inhibitors of NS5B polymerase of HCV won a prize at the Merck Research Laboratories Target-to-phase IIb Symposium in Atlanta. Since 2007, she has been working in Prof. A. Boffi’s group at “La Sapienza” University on a project focused on the redox metabolism of Leishmania infantum. In particular, a study on the trypanothione reductase disclosed the mechanism of inhibition of antimonials, widely used as a drug against leishmaniasis.



Was born in Varese in 1973 and graduated in Biological Sciences in 1997 at the University of Pavia. The scientific background of Claudia Binda is in the field of structural biology of medically-relevant proteins. Her research activity mostly developed at the University of Pavia in the Structural Biology group headed by Prof. Andrea Mattevi. For more than 10 years she has been collaborating with the group of Prof. Dale Edmondson at Emory University (USA), where she spent some time of her career for biochemical studies on monoamine oxidases supported by a fellowship from a NIH grant. More recently, she has been working on Lysine-specific Demethylase 1, the first histone lysine demethylase ever discovered, and on a new tuberculosis drug target. Since 2008 she has been Assistant Professor in Molecular Biology at the University of Pavia.




After completing her undergraduate studies in Biology at the University of Turin, moved to the National Human Genome Research Institute (NIH, USA). Her work ever since has been focused on gaining a better understanding of the pathogenesis of Primary Immunodeficiencies and on the development of gene therapy-based innovative therapeutic approaches for these diseases. At the NIH, she gained extensive experience in gene therapy with retroviral vectors, both in human cells and in the mouse model of IL12R1 deficiency. Since 2006 she has been working at the Telethon Institute for Gene Therapy in Milan, focusing on the evaluation of safety and efficacy of a hematopoietic stem cell-based gene therapy approach for Wiskott-Aldrich syndrome (WAS). Her work has contributed to the approval of a clinical trial of lentiviral vector-mediated gene therapy for WAS that started in 2010 at the San Raffaele Hospital in Milan and in which until now 4 WAS patients have been enrolled and successfully treated.



PhD, graduated in Pharmacy in 2001 at the Universita’ degli Studi in Milan. She received the PhD in Cellular and Molecular Biology in 2007 working at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET). After a period at the ITB-CNR laboratory of Milan, from 2010 to 2012, she was senior postdoctoral fellow at the Massachusetts General Hospital, Harvard Medical School. Since 2012 she has been a research scientist in the IRGB-CNR laboratory at the Humanitas Clinical Institute. Her activity focuses mainly in the field of genetic diseases, to which she hascontributed with the identification of the pathogenic mechanisms in different forms of primary immunodeficiencies and osteopetrosis. She was honored with scholarships from national and international organizations (Marie Curie, EMBO) and awards from several international scientific societies.




Graduated in Pharmaceutical Biotechnology in 2003 at the University of Padua and in Molecular and Cellular Biology in 2011 at the University of Bologna. Marta obtained her PhD in Biotechnology in 2007, at the University of Padua. During previous research experiences in the field of Biomedical Sciences, she worked on projects whose aim was to unravel the molecular mechanisms at the basis of some diseases (such as Huntington’s Corea, Alzheimer’s disease, deafness), with particular emphasis on the alterations of intracellular Ca2+ homeostasis and mitochondrial activity. Marta has gained skills and expertise in Molecular and Cellular Biology, as well as in Fluorescence Microscopy, since she had the opportunity (during her Ph.D and post-doctoral fellowships) to work in laboratories with a solid background in these techniques, such as the laboratory of Professors. T.Pozzan, E.Carafoli, B Cozzi (University of Padova, Italy), R.Y. Tsien (University of San Diego, CA) and Prof. L.Scorrano.

  • Ca2+ hot spots on the mitochondrial surface are generated by Ca2+ mobilization from stores, but not activation of store operated Ca2+ channels. Molecular Cell 2010; 38: 280-290.
  • Mitochondrial fission and cristae disruption increase the response of cell models of Huntington’s disease to apoptotic stimuli. EMBO Mol Med 2010; 2: 490-503



Was born in Rieti in 1980. He has a Masters Degree in Biological Sciences and a PhD in Genetics and Molecular Biology obtained at the University of Rome (2007). During his PhD he worked on gene therapy of Duchenne Muscular Dystrophy and studied microRNAs in hematopoiesis and myeloid leukemias. As a post-doc at the Rockefeller University in New York (2007-2010), he studied microRNAs in embryonic stem cells. From 2009 to 2010 he was director of the Rockefeller Stem Cells Derivation Core. Since 2011 he returned permanently in Italy, obtaining a Researcher position at the Sapienza University, to work on Amyotrophic Lateral Sclerosis with reprogrammed stem cells (iPS). His works have been published in Cell, Dev. Cell, EMBO J, PNAS and other important international journals. He was awarded with a HFSP Fellowship, a NY Stem Cell Found. Fellowship, the Young Researcher SIBBM 2006 Award and the RNA Society 2008 Poster Award.

  • A minicircuitry comprised of microRNA-223 and transcription factors NFI-A and C/EBPalpha regulates human granulopoiesis Cell 123, 819–831 (2005).
  • The miR-430/427/302 Family Controls Mesendodermal Fate Specification via Species-Specific Target Selection Dev Cell 16, 517–527 (2009).



After completing her degree in Biology at the University of Tuebingen in Germany and the University of Queensland in Australia, Aisha moved to Italy to pursue her PhD at the San Raffaele Telethon Institute for Gene Therapy. She has since become interested in studying the pathogenesis of primary immunodeficiencies and their treatment with innovative approaches, such as gene therapy. Her work is focused on adenosine deaminase (ADA)-deficient severe combined immunodeficiency, a fatal condition unless treated. She has identified specific alterations in immune and non-immune cells in ADA-deficiency, which can be corrected by gene therapy. During a period spent at Yale University in the US she has studied the efficacy of gene therapy on immune tolerance. With 18 ADA-SCID patients successfully treated in Milan, this disease nowadays represents a paradigmatic model of gene therapy for inherited disorders. Her current work at TIGET is focused on the market registration of this innovative therapy, in order to make it available to ADA-SCID patients worldwide.



Achieved her Masters in Biological Sciences in 2002 at the University of Naples Federico II and has continued her scientific activity leading to Ph.D. in Biotechnology in 2006 at the Second University of Naples. Currently she is a Telethon researcher. Her interest is in the field of human genetics and the study of causative mechanisms of rare congenital diseases. She has been studying the pathogenic mechanisms that underly the diseases caused by alteration in physiological dosage of genes regulated by genomic imprinting (Beckwith-Wiedemann syndrome, Silver-Russell syndrome and Wilms’ tumor). The results obtained have contributed to understanding how the genetic and epigenetic defects arise, are inherited and maintained in patients with growth alterations.



(14/2/77) Is a research fellow in the laboratory headed by Prof. Elena Cattaneo at University of Milan (Università degli Studi). She graduated in Biological Sciences (2001) and got her doctorate degree in Biotechnology applied to Pharmacology (2006) at the University of Milan. From the beginning of her career, her research has been focused on mechanisms of action of mutant huntingtin, the protein that causes Huntington’s disease, a rare neurodegenerative disease characterized by movement disorders, cognitive and behavior.
Marta Valenza has contributed to identifying a decrease in the production of brain cholesterol in Huntington’s disease, identifying the underlying molecular mechanism. This dysfunction – of cerebral origin but measurable in the blood of patients – has attracted the interest of clinics for therapeutics and biomarkers researches. In 2009, she was a visiting scientist at the Centre for Molecular Medicine and Therapeutics, Vancouver (Canada), in the laboratory of Prof. Michael Hayden, a scientific leader in the research on Huntington’s disease and lipids, and now president of Global Research and Development, TEVA Pharmaceutical Industries.



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