ft credits@ceinge
Biochemistry of Lysosomal Diseases

Luigi Michele Pavone


Full Professor of Biochemistry at the Department of Molecular Medicine and Medical Biotechnology, Medical School of the University of Naples Federico II.

Prof. Pavone research activity is based on the molecular basis of lysosomal diseases and in particular of Mucopolysaccharidosis. Author of more than 60 articles published in international scientific journals, Prof. L. Pavone has actively participated as a speaker at international and national conferences in the field of lysosomal diseases. Furthermore, he has been coordinator in numerous research studies funded by international, national, and regional agencies for the development of new therapeutic approaches for Mucopolysaccharidosis IIIB (Sanfilippo B Syndrome). Prof. Pavone attended relevant foreign university institutions such as the Baylor College of Medicine (Houston, Texas), INSERM U616 (Paris, France) and the MRC Laboratory of Molecular Biology (Cambridge, UK). Prof. Pavone is the author of patents for the treatment of Mucopolysaccharidoses. Prof. Pavone is also Head of the Laboratory of Biochemical Diagnostics of Lysosomal Diseases at CEINGE.

The activities of Prof. Pavone's research group are aimed at developing new therapies for lysosomal diseases many of which to date do not have a cure for the neurological defects of children affected by these pathologies. Furthermore, the laboratory deals with the development of new analytical techniques useful for the early diagnosis of lysosomal diseases.

Most relevant publications
  1. De Pasquale V, Esposito A, Scerra G, Scarcella M, Ciampa M, Luongo A, D'Alonzo D, Guaragna A, D'Agostino M, Pavone LM. N-Substituted L-Iminosugars for the Treatment of Sanfilippo Type B Syndrome. J Med Chem. 2023 Feb 9;66(3):1790-1808. doi: 10.1021/acs.jmedchem.2c01617.
  2. De Pasquale V, Scerra G, Scarcella M, D'Agostino M, Pavone LM. Competitive binding of extracellular accumulated heparan sulfate reduces lysosomal storage defects and triggers neuronal differentiation in a model of Mucopolysaccharidosis IIIB. Biochim Biophys Acta Mol Cell Res. 2021 Oct;1868(11):119113. doi: 10.1016/j.bbamcr.2021.119113.
  3. De Pasquale V, Pavone LM. Heparan sulfate proteoglycans: The sweet side of development turns sour in mucopolysaccharidoses. Biochim Biophys Acta Mol Basis Dis. 2019 Nov 1;1865(11):165539. doi: 10.1016/j.bbadis.2019.165539.
  4. De Pasquale V, Sarogni P, Pistorio V, Cerulo G, Paladino S, Pavone LM. Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses. Mol Ther Methods Clin Dev. 2018 Jun 18;10:8-16. doi: 10.1016/j.omtm.2018.05.002.
  5. De Pasquale V, Pezone A, Sarogni P, Tramontano A, Schiattarella GG, Avvedimento VE, Paladino S, Pavone LM. EGFR activation triggers cellular hypertrophy and lysosomal disease in NAGLU-depleted cardiomyoblasts, mimicking the hallmarks of mucopolysaccharidosis IIIB. Cell Death Dis. 2018 Jan 18;9(2):40. doi: 10.1038/s41419-017-0187-0.