2023 Fifth International Brainstorm Symposium

Beverly L. Davidson, PhD, serves at the Chief Scientific Strategy Officer at Children's Hospital of Philadelphia (CHOP), is the Director for Tryamond G. Perelman Center for Cellular and Molecular Therapeutics, and is Arthur B. Meigs Chair in Pediatrics at CHOP. Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction.

Professor Steve Finkbeiner, MD, PhD, is a Professor at the University of California, San Francisco (UCSF) in the Departments of Neurology and Physiology. He is also the Director of the Center for Systems and Therapeutics and a Senior Investigator at Gladstone Institutes. The long-term goal of his reach is to understand how neuronal activity leads to learning and memory. Memory defects occur in several neurological and psychiatric diseases, and his lab has developed cell-based models of Huntington’s disease, Parkinson’s disease, ALS, frontotemporal dementia, Alzheimer’s disease, and more. The lab strives to understand the neurobiology of disease well enough to design rational interventions and produce effective treatments. The limitations of conventional approaches led them to invent new tools, such as robotic microscopy, and to adapt artificial intelligence to unravel cause and effect in complex mechanisms and gain insights from data that elude comprehension by the unaided human brain.

Dr. Fujii is an Assistant Professor of Molecular Genetics & Microbiology and Center for NeuroGenetics at the University of Florida College of Medicine. He obtained Ph.D. degrees in RNA biochemistry and molecular biology. The research vision of Dr. Fujii is to molecularly understand mammalian development and homeostasis by utilizing a variety of interdisciplinary approaches from cutting edge biochemistry to in vivo animal models.

Christine Klein, MD, PhD was appointed Lichtenberg Professor at the Department of Neurology of Luebeck University in 2005, where her research has focused on the clinical and molecular genetics of movement disorders and its functional consequences. In 2009, Dr. Klein been appointed Schilling Professor of Clinical and Molecular Neurogenetics at the University of Luebeck and became Director of the newly founded Institute of Neurogenetics in 2013.

Richard Morimoto, PhD and their lab is interested in cell stress regulatory pathways and protein homeostasis (proteostasis) that ensures the stability of the proteome in health and when challenged by cell stress and the expression of metastable, misfolded, and aggregated proteins. Using C. elegans and human iPS cells, we employ genetic, molecular, small molecule, proteomic and genomic methods and systems level analyses to identify changes during aging and age-associated diseases including metabolic diseases, cancer, and neurodegeneration.

Diane E. Merry, PhD is a professor in the department of Biochemistry and Molecular Biology at Thomas Jefferson University. The research in their lab centers on two areas of investigation to understand the molecular pathogenesis of the neurodegenerative disease spinal and bulbar muscular atrophy, which is caused by polyglutamine expansion within the androgen receptor (AR).

Dr. Nguyen is the newest member of the Center for Neurogenetics here at the University of Florida. Lien has a long-term interest in understanding molecular mechanisms and developing therapeutics for neurodegenerative disorders. Their research has focused on studying the roles of repeat expansion mutations in disease including Alzheimer's disease, Amyotrophic lateral sclerosis and Frontotemporal dementia.

Michael S. Okun is a neurologist, neuroscientist and author. He is the co-founder and director of the Norman Fixel Institute for Neurological Diseases at the University of Florida and is the chair of the Department of Neurology at the University of Florida (2015-current) and the Medical Director/Advisor for the Parkinson’s Foundation (2006-current). He is best known for his work in translating physiological principles underpinning human diseases, such as Tourette syndrome, tremor and Parkinson’s into real-world therapies.

Christopher Pearson, PhD, is a Professor of Molecular Genetics at the University of Toronto and a Senior Scientist in Genetics and Genome Biology at The Hospital for Sick Children. The Pearson lab research program builds upon the theme initiated over two decades ago to understand the disease-causing mutations of trinucleotide repeat expansions, that lead to diseases like Huntington’s disease and myotonic dystrophy.

Dr. Laura Ranum is the Director of the Center for Neurogenetics here at UF. She is also a Kitzman Family Professor of Molecular Genetics and Microbiology. Dr. Ranum’s lab focuses on understanding the fundamental mechanisms and developing therapies for repeat expansion disorders and other neurodegenerative diseases. Her lab discovered Repeat Associated Non-AUG (RAN) translation, a novel process that results in the production and accumulation of toxic proteins in patient tissues, including the brain and central nervous system.

Maurice S Swanson, PhD is a Professor and Associate Program Director for Center for NeuroGenetics here at UF. The Swanson lab studies the regulation of RNA processing during mammalian development and aging and how this regulation is altered in disease. Our research focuses on three main areas: 1) the roles of genomic repetitive elements in embryonic and postnatal development; 2) novel pathomechanisms of neurological diseases caused by the expansion of short tandem repeats (STRs); 3) RNA-based mechanisms of enhanced tissue repair in a mammalian model of optimized regeneration.

Dr. Stephen Tapscott studies how genes are turned on and off and how this activity goes awry in certain diseases. He focuses on the activity of certain “master” genes that control the development of muscle and nerve cells. He is a leader in understanding how the normal process of cell development is altered in two diseases: muscular dystrophy, a class of inherited diseases in which muscles weaken and atrophy over time; and rhabdomyosarcoma, a type of cancer that arises from muscle cells.

Leslie Thompson, PhD, has been committed to Huntington’s Disease Research for over 20 years, beginning with the collaborative search for the disease gene. They are also participant in the Venezuela Huntington’s Disease research group.

Dr. Eric Wang is an Associate Professor in the Center for NeuroGenetics here at UF. Dr. Wang's lab is focused on three main areas – studying the pathogenesis of microsatellite repeat diseases, in particular myotonic dystrophy, studying the basics of how RNA is processed and localized in cells in tissues, and combining insights made in both of those areas to develop treatments for people with these diseases.

Michael E. Ward, PhD research focuses on cellular and molecular mechanisms of frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS), two related neurodegenerative disorders. They use a combination of cell biology, proteomic, and functional genomic approaches in iPSC neuron models of ALS/FTD, with a long-term goal of understanding how disease-associated familial mutations lead to neurodegeneration.

Smita Saxena, PhD research focuses on identifying cellular mechanisms causally involved with onset and speaking of neurodegenerative diseases. They work with Identification and functional analysis of disease-related mechanisms involved in spinocerebellar ataxia1 and motoneuron disease.

Katrin Karbstein, Ph.D., is professor of Integrative Structural and Computational Biology at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology. Her research focuses on mechanisms of ribosome assembly, quality control and the molecular basis for ribosome-associated diseases (ribosomopathies).

Dr. Emily Plowman is a Professor at the University of Florida in the Departments of Speech, Language and Hearing Sciences, Surgery, and Neurology. She established and directs the Aerodigestive Research Core (ARC) whose mission is to improve assessment and clinical management approaches for upper aerodigestive tract disorders impacting communication, swallowing, and breathing function via pragmatically-guided clinical research.

Matthew LaVoie, PhD is the Fixel Family Chair, Co-Director Center of Translational Research in neurodegenerative disease at the University of Florida. The overall goal of the LaVoie lab is to elucidate the earliest molecular events responsible for adult-onset neurodegenerative diseases. LaVoie lab's focus on familial Parkinson’s disease is centered on pathogenic mutations in the Parkin and LRRK2 genes.

Dr. James Wymer is a neurologist and professor practicing at The University of Florida in Gainesville, Florida. He specializes in the diagnosis and management of neuromuscular diseases including amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, muscles diseases and peripheral neuropathies. Dr. Wymer has an extensive experience in clinical research, and has been the principal investigator in numerous clinical trials including studies of ALS, diabetic neuropathy, multiple sclerosis, migraine headaches and epilepsy.

Dr. Berglund is a professor at State University of New York at Albany and also the director of the RNA Institute. Dr. Berglund began studying biochemistry in 1990 with a focus on the structures that RNA can adopt and their role in biology. His interests expanded to determining the mechanisms through which RNA binding proteins recognize RNA motifs in pre-mRNA splicing.

Gary J. Bassell, Ph.D. joined the faculty at Emory University School of Medicine in 2005, where he is currently Professor and Chair of the Department of Cell Biology. Dr. Bassell’s laboratory at Emory has major interests to elucidate basic mechanisms of neuronal mRNA regulation and synapse development, and to understand neurological disease mechanisms that result from impairments in mRNA regulation, including fragile x syndrome and spinal muscular atrophy.