2023 Fifth International Brainstorm Symposium

The Center for NeuroGenetics at the University of Florida is pleased to announce the Fifth International Brainstorm Symposium


 January 25-27, 2023

Samuel P. Harn Museum of Art

 3259 Hull Road, Gainesville, FL 32611

About the Symposium

This two and a half-day symposium will bring together experts from around the world to discuss the role of repetitive sequences in disease.  In addition to microsatellites, the meeting will also focus on the role of other repetitive elements in biology and disease including repeat instability, repetitive sequences throughout the genome, Alu sequences, transposons, RNA and RAN proteins.

Harn Museum of art

Scientist and students are invited to attend our fifth international brainstorm on January 25th through the 27th. This year’s Symposium is going to be held at the Samuel P. Harn Museum of Art. We will be hearing from different scientific leaders in the field in all different concentrations and areas. There will also be abstract sessions from top trainees in the field.

This symposium aggregates outstanding and diverse group of scientists to discuss various aspects of genome biology and neurodegeneration. We have speakers and attendees from across the US, Canada, and Germany.

Our last international brainstorm was in 2020 and we are very excited to be hosting our 5th symposium. Previously this meeting inspired and connected young and more established researchers and has stimulated collaborations and interdisciplinary research. We hope to accomplish this again and continue to grow and expand our knowledge in neurogenerative diseases and repeat expansion diseases.


 Registration is closed. Questions? Contact centerforneurogenetics@health.ufl.edu

Confirmed Speakers

Bev Davidson


Beverly L. Davidson, PhD

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.


Director, Center for Systems and Therapeutics & Taube/Koret Center for Neurodegenerative Disease, Gladstone Institutes Professor, Departments of Neurology and Physiology University of California, San Francisco

Steve Finkbeiner, MD, PhD

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.


Assistant Professor, University of Florida

Kotaro Fujii, PhD

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.


Head of Institue of Neurogenetics, University of Lubeck

Christine Klein, MD, PhD

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.

Rick Morimoto

Professor, Northwestern

Richard Morimoto, PhD

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 Merry

Professor, Department of Biochemistry and Molecular Biology Vice Chair, Faculty Development and Engagement

Diane E. Merry, PhD

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).


Research Assistant Professor, University of Florida

Lien T. Nguyen, PhD

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 Okun

Chair And Professor of Neurology, University of Florida

Michael S Okun, MD

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

Senior Scientist, Genetics & Genome Biology, The Hospital for Sick Children

Christopher Pearson, PhD

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.


Kitzman Family Professor Of Molecular Genetics And Microbiology; Director Center For NeuroGenetics

Laura P W Ranum, PhD

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.


Professor And Associate Program Director, Center for NeuroGenetics, University of Florida

Maurice S Swanson, PhD

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.


Professor Human Biology Division, Fred Hutch; Professor Clinical Research Division, Fred Hutch

Stephen Tapscott, MD, PhD

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

Professor UCI, Sue & Bill Gross Stem Cell Research Center

Leslie Thompson, PhD

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.


Associate Professor, Center for Neurogenetics, University of Florida

Eric Wang, PhD

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 Ward

Investigator, Neurogenetics Branch, NINDS/NIH

Michael E. Ward, PhD

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

Department of Neurology, Inselspital

Smita Saxena, PhD

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

Professor, UF Scripps

Katrin Karbstein, PhD

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).


Professor and Doctoral Program Director, Aerodigestive Research Core, UNIVERSITY OF FLORIDA

Emily K Plowman, PhD, CCC-SLP

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.


Fixel Family Chair, Co-Director Center Of Translational Research In Neurodegenerative Disease, Associate Chair For Research-Neurology, UF

Matthew J LaVoie, PhD

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.


Professor & Division Chief; Melvin Greer Professor

James Wymer, MD, FAAN

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.

Professor, Deputy Director McKnight Brain Institute

Jada Lewis

Jada Lewis, Ph.D. is the deputy director of the MBI. Dr. Lewis is also a professor in UF’s department of neuroscience and investigator at the Center for Translational Research in Neurodegenerative Disease. She has co-led the MBI’s Education and Outreach Committee while also serving as a member of the MBI Executive Committee.

Andy Berglund

Professor, Director of The RNA Institute Biological Sciences, State University of New York at Albany

Andy Berglund, PhD

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 Bassell

Professor and Chair, Emory Univesity School of Medicine

Gary Bassell, PhD

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.

Travel and Accommodations Information

Nearest Airport: Gainesville Regional Airport
Hotel Information
Location of Symposium: Samuel P. Harn Museum of Art

Symposium Schedule

Day One | January 25th, 2023

Time Speaker Topic
6-7pm Registration and Check-In
Session 1 Keynote ––
7:15-7:20pm David Nelson, PhD Welcome Message
7:20-7:25pm Azra Bihorac, MD, MS, FASN, FCCM Welcome Message
7:25-7:30pm Laura Ranum, PhD Welcome and Symposium Overview
7:30-8:30pm Beverly Davidon, PhD Therapeutic approaches for repeat expansion diseases using AAV (keynote)
8:30-8:55pm Discussion

Day Two | January 26th, 2023

Time Speaker Topic
Session 2 –– ––
8-8:30am Leslie Thompson, PhD A systems approach to understanding pathogenesis in HD
8:30-9am Diane Merry, PhD Spinal bulbar muscular atrophy: USP7 a novel mechanism and potential therapeutic target
9-9:30am Rick Morimoto, PhD Polyglutamine toxicity accelerates proteostasis collapse in aging.
9:30-10am Coffee Break & Photos
Session 3
10-10:30am Maury Swanson, PhD RNA gain of function signatures in neurological disease
10:30-11am Andy Berglund, PhD Evaluating the molecular response to small molecule or exercise interventions in myotonic dystrophy
11-11:30am Eric Wang, PhD Repeat expansions institute supply chain issues in the nucleus and cytoplasm.
11:30am-12pm Gary Bassell, PhD Regulation of RNA granules, local translation and membrane trafficking by the fragile x messenger ribonucleoprotein-1 (FMRP)
12-1:20pm –– Lunch and Poster Session
Session 4 ––
1:20-1:50pm Michael Ward, PhD Mining the proteome for biomarkers in ALS and other neurodegenerative disorders
1:50-2:20pm Jada Lewis, PhD Clinical and mechanistic principles of Alzheimer’s disease
2:20-2:50pm Christopher Peason, PhD Single-stand DNA binding proteins as modifiers of repeat instability and disease
2:50-3:30pm Poster Talks ––
3:30-4pm –– Coffee Break
Session 5 ––
4-4:30pm Laura Ranum, PhD Repeat associated non-AUG proteins in neurodegenerative disease: mechanistic insights and therapeutic opportunities.
4:30-5pm Smita Saxena, PhD Unravelling cellular pathology in C9orf72-ALS
5-5:30pm Lien Nguyen, PhD Detection and isolation of a novel repeat expansion mutation for AD
5:30pm Announcements Arrangements for Transportation to Gala Dinner
6-9pm Gala Dinner

Day 3 | January 27th, 2023

Time speaker Topic
Session 6 –– ––
8-8:30am Jim Wymer, MD, FAAN Clinical assessments and trials in Amyotrophic lateral sclerosis
8:30-9am Emily Plowman, PhD, SLP-CCC Staying Ahead of the Curve: proactive therapeutics to optimize breathing, airway clearance & swallowing in neurodegenerative disease
9-9:30am Michael Okun, PhD Deep brain stimulation: Parkinson’s and beyond
9:30-10:10am Poster Talks
10:10-10:40 Coffee Break
Session 7
10:40-11:10am Christine Klein, PhD Genetic modifiers in X-linked Dystonia Parkinsonism (XPD)
11:10-11:40am Matthew LaVoie, PhD Intersecting pathways in Parkinson’s Disease
11:40am-12:10pm Steve Finkbeiner, PhD How can artificial intelligence help us understand mechanisms and find therapies for triplet repeat diseases?
12:10-1:20pm Lunch and Poster Session
Session 8
1:20-2pm Poster Talks
2-2:30pm Katrin Karbstein, PhD Ribosome quality control during and after assembly
2:30-3pm Kotaro Fujii, PhD Mechanisms of translational regulation and proteome diversity
3-3:20pm Coffee Break
Session 9 Keynote
3:20-4:20pm Stephen Tapscott, PhD, MD Molecular mechanisms of FSHD (keynote)
4:20-4:40pm Discussion
4:40-4:55pm Eric Wang, PhD Short Talk Awards
4:55-5pm Laura Ranum, PhD Closing Remarks

Poster Session

Poster Sessions will be at 12-1:20pm on January 26th and 12:10-1:20pm on January 27th.


Maurice S Swanson

Maurice S Swanson

Professor And Associate Program Director


(352) 273-8076

Laura P W Ranum

Laura P W Ranum

Kitzman Family Professor Of Molecular Genetics And Microbiology; Director Center For NeuroGenetics


(352) 294-5209

Eric Wang

Eric Wang

Associate Professor


(352) 273-7601

Kotaro Fujii

Kotaro Fujii

Assistant Professor


(352) 273-7279

Lien T Nguyen

Lien T Nguyen

Assistant Professor


(352) 273-5847


The Center for NeuroGenetics is very thankful for our sponsors who generous supported the 5th International Brainstorm Symposium.

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