IRCND

Dr. Leonard Petrucelli, PhD is the Ralph B. and Ruth K. Abrams Professor of Neuroscience at the Mayo Clinic in Jacksonville, FL, where his lab works on understanding the molecular mechanisms that underly abnormal protein aggregation in neurodegenerative diseases. Dr. Petrucelli and his research team are constantly at the forefront of their field, researching the mechanisms that cause degeneration in Alzheimer's disease, amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD) and harnessing their findings into the development of novel therapeutic targets and biomarkers.

Seminal findings from his team include the discovery of a hallmark of C9orf72-linked FTD and ALS (c9FTD/ALS): c9RAN proteins (dipeptide repeats). These proteins result from a mutation in the C9orf72 gene. His team is investigating the significance of c9RAN proteins in various cell models, including patient-derived fibroblasts, and in vivo systems. His lab also recently discovered that the enzyme HDAC6 may be an effective strategy to effectively arrest the toxic accumulation of tau protein, which is associated with Alzheimer's disease and related dementia. Dr. Petrucelli's team has also shown that detection of c9RAN proteins in cerebrospinal fluid of patients carrying the C9orf72 mutation holds promise as a potential biomarker. More recently, his group has identified a novel tau protein that can be detected in Alzheimer's disease patients.

Professor Chiara Zurzolo, MD, PhD, is head of the Membrane Trafficking and Pathogenesis Unit and Director of the Brain connectivity and neurodegenerative diseases research program at the Institut Pasteur (Paris), where she studies the mechanisms of protein trafficking in health and diseases with a focus on neurodegenerative diseases and brain tumors.

She received her MD and then PhD in molecular and cell biology from the University of Naples Federico II. She then obtained a postdoctoral fellowship to spend four years at Cornell Medical School, New York where she uncovered some of the mechanisms of apical protein trafficking in polarized epithelial cells. In 1995, she returned to Naples to establish her laboratory at the Faculty of Medicine and Surgery at Naples University Federico II as a professor of Cell Biology.

In 2015, she was elected member of the European Molecular Biology Organization (EMBO) and promoted Professor at Institut Pasteur. In 2019, she was a visiting scholar at Harvard University, sponsored by the Radcliffe Fellowship. Her international reputation leads her to conferences and seminars all over the world. She is also very involved in teaching and organizing national/international conferences and courses.

Dr. Don W. Cleveland is the Distinguished Professor and Chair of the Department of Cellular and Molecular Medicine at University of California San Diego. Dr. Cleveland is a cancer biologist and neuroscientist that has made ground-breaking contributions in the regulation of assembly of mitotic spindles and chromosome movement. He discovered the microtubule associated protein tau (mutation in which causes human cognitive disease), the tubulin gene families encoding the major subunits of microtubules, and the first mammalian example of control of gene expression through regulated RNA instability.

Dr. Cleveland showed that disorganization of neurofilaments causes selective failure of motor neurons in mice and humans. He then demonstrated that similar disease could also arise by a toxicity of mutant superoxide dismutase unrelated to its normal activity, thereby uncovering the mechanism underlying a major genetic form of Amyotrophic Lateral Sclerosis (ALS). He also showed that motor neuron death in inherited ALS is non-cell autonomous, requiring mutant damage to both motor neurons and the neighboring supporting cells. This discovery has wide implications for other major neurodegenerative diseases.