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个人简介
Pasquale Piccolo graduated in Medical Biotechnology in 2005 from University of Naples “Federico II” and then completed a Ph.D. in Human Genetics, also at the University of Naples in 2009. He then carried out post-doctoral research in Philip Ng’s lab at Baylor College of Medicine in Houston TX, here he became interested in liver-directed gene therapy with helper-dependent adenoviral vectors. He then returned to Italy, moving to TIGEM to work in Nicola Brunetti-Pierri’s lab. Here he investigated the role of transcription factors and microRNAs in the pathogenesis of liver disease related to α1-antitrypsin deficiency. Given the importance of this work, he was awarded with the α1-Antitrypsin Laurell's Training Award and the Gordon L. Snider Scholar Award. His research has also explored the potential of small molecules for the treatment of inherited diseases; he identified losartan as an effective treatment for the extracellular matrix deposition defect of Myhre syndrome, a rare connective tissue disease. Losartan has been shown to ameliorate ECM deposition in Myhre patients in a recent clinical trial. Since his appointment as an independent assistant investigator at TIGEM, his research has been focused on the development of novel liver-directed gene therapy approaches for genetic disorders with liver damage.
研究兴趣
论文共 64 篇作者统计合作学者相似作者
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MOLECULAR THERAPYno. 4 (2023): 493-493
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Nunzia Pastore,Francesco Annunziata, Rita Colonna,Veronica Maffia,Teresa Giuliano, Bruno Maria Custode,Bernadette Lombardi,Elena Polishchuk,Vincenzo Cacace,Lucia De Stefano,Edoardo Nusco,Nicolina Cristina Sorrentino,
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P. Piccolo,R. Ferriero, C. Perna, M. Monti, R. De Cegli,A. Barbato,E. Nusco, S. Campione,N. Brunetti-Pierri
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HUMAN GENE THERAPYno. 23-24 (2022): A170-A170
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HEPATOLOGY COMMUNICATIONSno. 12 (2022): 3597-3597
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