Additionally, here are two useful articles about HD and sleep written by Dr. Morton: 1) HD and Sleep and 2) Simple rules for a good night's sleep in Huntington's disease.

About the Speakers

A. Jennifer Morton, PhD, ScD, FRSB
Professor of Neurobiology
University of Cambridge
Professorial Fellow
Director of Studies in Medicine and Veterinary Medicine
Newnham College, Cambridge

Dr. A. Jennifer Morton has been working on Huntington’s disease ever since she set up her laboratory at the University of Cambridge in 1991. She is interested in understanding the relationship between neurodegeneration and the neurological symptoms in HD. She is particularly interested in sleep, circadian rhythms and cognitive decline in HD. With funding from the Hereditary Disease Foundation, The Wellcome Trust, and CHDI Foundation, she has characterized the behavioral profile of a number of mouse models of HD.

For the past 10 years, Dr. Morton has also been working with HD sheep. She believes that, although it is an unconventional model, there is much to be gained from understanding the behavioral pathology in this large-brained diurnal model of HD. Her lab’s goal is to identify quantifiable measures of behavior that can be used to test the efficacy of novel therapies for HD.

H. Diana Rosas, MD
Associate Professor, Departments of Neurology and Radiology
Director, Center for Neuro-imaging of Aging and Neurodegenerative Disease
Massachusetts General Hospital, Harvard Medical School

Dr. H. Diana Rosas focuses primarily on the development of biomarkers for use in the study of neurodegenerative diseases to better characterize progression, to better understand genotype/phenotype correlations, and to apply novel neuro-imaging approaches in clinical trials with the overall aim of making the trials more efficient.

Dr. Rosas and her team have begun to develop models that may explain clinically heterogeneous phenotypes and variability in disease progression. They plan to expand their efforts to include several different types of imaging approaches that promise more precise measurements and may provide novel and important information on the neural underpinnings of HD and their clinical consequences.

Thank you to our webinar sponsor!

About the Speakers

Osama Al-Dalahmah, MD, PhD
Department of Neuropathology
Columbia University Irving Medical Center, New York-Presbyterian Hospital

Dr. Osama Al-Dalahmah’s research is focused on understanding how the brain's glial cells, the supportive cells that maintain the function of other brain cells, become abnormal in neurodegenerative diseases, especially Huntington’s disease. Ultimately, the goal of his research is to find ways to modify the function of glial cells in order to protect the brain from damage and slow down the progression of the neurodegeneration.

Shawn Davidson, PhD
Lewis-Sigler Institute for Integrative Genomics
Princeton University

Dr. Shawn Davidson’s research is at the interface of biotechnology and genetics. His lab studies various diseases using imaging mass spectrometry – a technique that allows him to pinpoint where within the brain certain proteins are expressed. He combines understanding of these expression changes with disease-related pathology and mathematical modeling for the development of therapeutic strategies.

Thank you to our webinar sponsors!

About the Speakers

Michelle Gray, PhD
University of Alabama at Birmingham

Dr. Michelle Gray is an Associate Professor in Neurology at the University of Alabama at Birmingham (UAB). Her laboratory is supported by grants from NIH/NINDS and the Hereditary Disease foundation. She uses a mouse model that that she developed in the lab of Dr. William Yang during her postdoctoral studies at the University of California, Los Angeles to understand the contribution of specific cell types to the pathogen edits of Huntington’s disease. Although she has a new interest in the role of mHTT in the cardiovascular abnormalities in HD, her laboratory has been primarily focused on non-neuronal cells in the brain, specifically astrocytes and their contribution to HD pathogenesis.

Sophie St-Cyr, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania

Dr. Sophie St-Cyr is currently a postdoctoral fellow in Dr. Beverly Davison’s lab in the Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, Philadelphia, PA. She obtained a Master’s degree from the University of Montreal and completed a PhD on the long-term impacts of prenatal stress at the University of Toronto in 2017. Dr. St-Cyr developed an interest to explain the molecular mechanisms underlying phenotypic changes. Currently, her research focuses on the heart pathology in Huntington’s disease to improve the quality of life of patients. She explores the contribution of changes in gene and microRNA expression in the HD reduced heart size and contractile function. 

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About the Speaker

Steven Finkbeiner, MD, PhD
Gladstone Institutes
University of California, San Francisco
2022 Leslie Gehry Prize for Innovation in Science Recipient

Dr. Steven Finkbeiner is interested in understanding mechanisms of neurodegenerative disease and developing therapeutic strategies and therapies. He has studied HD since 1998, inventing and applying innovative methods and tools, including robotic microscopy, stem cells and artificial intelligence. His landmark study in 2004 in Nature changed the way the field thought about the hallmark pathology in Huntington’s disease, the most cited paper in neuroscience for the decade. With philanthropists in the San Francisco Bay Area, he established the Taube-Koret Center, which works to translate the most promising discoveries from the labs into therapeutics, often in partnership with drug companies. His most promising HD therapeutic strategy stimulates a pathway in cells that helps clear the abnormal Huntingtin protein.

Thank you to our webinar sponsor!

Join us as leading scientists welcome us into their labs to share the latest in Huntington's disease research and how it moves from the laboratory to drug development.

Learn from HDF-funded researchers about what motivates and excites them - they are the future of HD research!

Meet a family living with Huntington's disease who will share their hope in the research the Foundation funds.

Participate in “Ask the Scientists” at the end of the program.

Celebrate the extraordinary science that is changing the face of Huntington's disease!

The Hereditary Disease Foundation Gala 2022 is open to all free of charge.

Thank you to our Sponsors!

Bronze Level

About the Speakers

Michelle Gray, PhD
University of Alabama at Birmingham

Dr. Michelle Gray is an Associate Professor in Neurology at the University of Alabama at Birmingham (UAB). Her laboratory is supported by grants from NIH/NINDS and the Hereditary Disease foundation. She uses a mouse model that that she developed in the lab of Dr. William Yang during her postdoctoral studies at the University of California, Los Angeles to understand the contribution of specific cell types to the pathogen edits of Huntington’s disease. Although she has a new interest in the role of mHTT in the cardiovascular abnormalities in HD, her laboratory has been primarily focused on non-neuronal cells in the brain, specifically astrocytes and their contribution to HD pathogenesis.

Ryan Lim, PhD
University of California, Irvine

Dr. Ryan Lim is the Director of Biology at Modulo Bio, Inc., and works with the lab of Dr. Leslie Thompson at the University of California, Irvine as a member of the NeuroLINCS and AnswerALS consortia. He has 10+ years of experience studying neurological disorders using patient-derived stem cells and mouse models and established the first induced pluripotent stem cell-derived Blood-Brain Barrier model of neurodegeneration, which was Huntington's disease. He was at the forefront of using single cell technologies and has expertise in genomics and systems/computational biology applications, and has research awards from CIRM, American Heart Association, Hereditary Disease Foundation, and the NIH.

Ricardo Mouro Pinto, PhD
Massachusetts General Hospital, Harvard Medical School, Boston

Dr. Ricardo Mouro Pinto is an Instructor in Neurology at the Center for Genomic Medicine, Massachusetts General Hospital and Harvard Medical School. He is an applied human geneticist and neuroscientist with a strong interest in unstable microsatellite-associated neurodegenerative disorders, such as Huntington's disease and Friedreich Ataxia. A major focus of his Lab has been the characterization of the mechanisms responsible for DNA repeat instability in the context of such diseases and as potential therapeutic targets. Dr. Mouro Pinto is the inaugural recipient of HDSA's Berman-Topper HD Career Development Fellowship.

Sarah Hernandez, PhD, Director of Research Programs
Hereditary Disease Foundation

Dr. Sarah Hernandez oversees the Foundation’s scientific research portfolio and works closely with its Scientific Advisory Board to identify the most promising research. 

Dr. Hernandez learned at a young age that her family was affected by Huntington's disease, motivating her to learn more and inspiring her to become a scientist. In 2015, she received a PhD in Biology and Biotechnology from Worcester Polytechnic Institute. For her postdoctoral studies, she joined the lab of renowned Huntington’s disease scientist Leslie Thompson, PhD, at the University of California, Irvine and Chair-Elect, HDF Scientific Advisory Board. There she worked on understanding the molecular consequences of Huntington’s disease to cells of the brain using stem cells and fruit flies. In 2021, she was honored as the recipient of the Hereditary Disease Foundation Nancy S. Wexler Young Investigator Prize, presented to a researcher whose work represents the highest caliber of excellence, diligence, and creative thinking. Additionally, Sarah is an editor for HDBuzz, writing articles about Huntington’s disease-related science and research news in plain language for the patient community.

About the Speakers

Leslie M. Thompson, PhD
University of California, Irvine
2013 Leslie Gehry Brenner Prize for Innovation in Science Recipient

Dr. Leslie Thompson has studied Huntington’s disease for most of her scientific career. She was a member of the HDF’s Venezuela HD Project that identified the causative gene for HD in 1993. She is trying to understand how the HD mutation damages brain cells and identify targets for new drugs to prevent or ameliorate the damage. She is also looking at how the mutation influences modifications of the huntingtin protein and other cellular molecules. In addition, Dr. Thompson worked with a group of investigators to establish the HD patient-derived iPS cell consortium (induced pluripotent stem cells) and is using stem cells to study HD through multi-institutional collaborations and Big Data approaches. She is currently evaluating the use of human neural stem cells as a possible therapy for HD.

Steven Finkbeiner, MD, PhD
Taube/Koret Center for Neurodegenerative Disease
Gladstone Institutes
University of California, San Francisco

Dr. Steven Finkbeiner is interested in understanding mechanisms of neurodegenerative disease and developing therapeutic strategies and therapies. He has studied HD since 1998, inventing and applying innovative methods and tools, including robotic microscopy, stem cells and artificial intelligence. His landmark study in 2004 in Nature changed the way the field thought about the hallmark pathology in Huntington’s disease, the most cited paper in neuroscience for the decade. With philanthropists in the San Francisco Bay Area, he established the Taube-Koret Center, which works to translate the most promising discoveries from the labs into therapeutics, often in partnership with drug companies. His most promising HD therapeutic strategy stimulates a pathway in cells that helps clear the abnormal Huntingtin protein.

About the Speaker

Elena Cattaneo, PhD, OMRI
University of Milan
2021 Leslie Gehry Brenner Prize for Innovation in Science Recipient

Dr. Elena Cattaneo leads a laboratory at the University of Milan focused on understanding the physiopathology of Huntington’s disease. Her goal is to develop pharmacological, genetic, and cellular strategies that slow down the course of HD or prevent its onset. She is director of the Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases at the Department of Biosciences. Her laboratory works with HD clinical experts in Italy and globally to assess the diagnostic and therapeutic potential of research findings for clinical translation and application. She is also the co-founder and director of UniStem, the University of Milan’s coordinated center on stem cells which promotes access to information on the study of stem cells to a network of over 100 universities and research centers internationally. In 2013, Professor Cattaneo was appointed a Senator for Life by the President of Italian Republic in recognition of her activities as a champion of science.

About the Speakers

Beverly L. Davidson, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania
2015 HDF’s Leslie Gehry Brenner Prize for Innovation in Science Recipient

Dr. Davidson’s laboratory focuses on genetic diseases that affect the brain, investigating how mutant gene products contribute to disease, and why certain brain regions are more susceptible than others. Her team employs advanced molecular methods, sequencing and imaging in animal models, and a variety of molecular tools to test various hypotheses. Her lab is also developing next generation therapeutics for inherited disorders, including the engineering of novel gene therapy vector capsids and cargo to approach tissue and cell type specific treatments.

Alejandro Mas Monteys, PhD
Children’s Hospital of Philadelphia
University of Pennsylvania

Dr. Mas Monteys is developing and testing a treatment for Huntington's disease using a novel allele specific CRISPR/Cas9 gene editing approach to eliminate expression of the abnormal protein causing HD. This technique offers an exciting extension of gene editing applications for HD and has significant implications for other human diseases.

The focus of Dr. Mas Monteys’s research and his unique expertise will continue to advance our understanding of the mechanisms of gene regulation by providing tools and technological methods to researchers across the world.

About the Speaker

About the Speaker

Join us to hear an update on current clinical trials on Huntington's disease. Learn about the groundbreaking approaches to treatments. And meet HD families, our Partners in Research.

This virtual event will take us into the laboratories of several of our leading scientists to hear about today’s most promising, cutting-edge research.

Dr. Francis Collins, Director of the National Institutes of Health, will present our 2021 Leslie Gehry Brenner Prize for Innovation in Science and 2021 Nancy S. Wexler Young Investigator Prize.

Celebrate the brilliant science that is changing the face of Huntington's disease!

The Hereditary Disease Foundation Gala 2021 is open to all free of charge.

Clinical Trials Update and Research Pathways

&

Ask the Scientists

Andrew Feigin, MD
Chair, Huntington Study Group
Professor, Department of Neurology at NYU Grossman School of Medicine
Executive Director, The Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders
Associate Chair for NYU Network and Clinical Research, Department of Neurology

Sarah Hernandez, PhD
Hereditary Disease Foundation Postdoctoral Fellowship Recipient
Postdoctoral Fellow, Laboratory of Leslie Thompson, PhD, University of California, Irvine
Editor, HDBuzz

2021 Awards

Presented by Francis S. Collins, MD, PhD
Director, National Institutes of Health (NIH)

2021 Leslie Gehry Brenner Prize for Innovation in Science
Elena Cattaneo, PhD, OMRI, University of Milan

2021 Nancy S. Wexler Young Investigator Prize
Sarah Hernandez, PhD, University of California, Irvine

Thank you to our Sponsors!

and

About the Speakers

Brent Fitzwalter, PhD
Massachusetts Institute of Technology, Cambridge, MA

Outfoxing HD: Does the Aging-Related Gene FOXO3 Hold the Key?

Brent Fitzwalter is a Hereditary Disease Foundation Postdoctoral fellow in the laboratory of Dr. Myriam Heiman at Massachusetts Institute of Technology in Cambridge, MA. Brent completed his Bachelor’s degree in Biochemistry at the University of Missouri-Columbia. He obtained his PhD in Pharmacology at the University of Colorado studying the molecular mechanisms that link autophagy and cell death. Currently, his research in Dr. Heiman’s lab focuses on the role of the aging-related transcription factor FOXO3 in Huntington’s disease.

Rachel Harding, PhD
University of Toronto, Canada

Unlocking the Secrets of the Mysterious HD Protein

Rachel Harding is a HDSA Berman Topper fellow in the lab of Professor Cheryl Arrowsmith at the Structural Genomics Consortium (SGC) at the University of Toronto, Canada. Rachel completed both her undergraduate and DPhil at the University of Oxford where she trained in structural biology and protein chemistry. Rachel is researching the Huntington’s disease protein, huntingtin, and investigating its structure-function relationship.

About the Speaker

Christopher E. Pearson, PhD
Senior Scientist, Program of Genetics & Genome Biology
The Hospital for Sick Children
Full-Professor, Department of Molecular Genetics
University of Toronto
Canada Research Chair in Disease-Associated Genome Instability

Since 1993 Christopher Pearson has studied the mechanisms of disease-causing repeat instability associated with neurological, neurodegenerative and neuromuscular disease, with the goal of developing treatments by targeting the disease-causing repeat expansions. His lifetime goal is to treat repeat diseases by arresting or reversing somatic CAG expansions.

His lab works on molecular, cellular, and mouse models, and patient tissues of Huntington’s disease, myotonic dystrophy, various spinocerebellar ataxias, fragile X mental retardation, and C9orf72-associated amyotrophic lateral sclerosis. Focus is upon DNA repair, DNA damage, epigenetics, and unusual DNA structures formed by the repeats. Recent advances include identifying repeat expansions associated with autism, as well as identifying the first small-molecule to induce contractions of the expanded CAG repeat in the striatum of HD mice.

About the Speaker

Ai Yamamoto, PhD
Associate Professor of Neurology, Columbia University
Recipient, 2020 Leslie Gehry Brenner Prize for Innovation in Science

Dr. Ai Yamamoto made a stunning entrance into the field of Huntington's disease research while she was still a graduate student. She demonstrated that Huntington's disease might be reversible — something no one had shown up to that point. Dr. Yamamoto's work led her and her research team to a fascinating protein called Alfy which has connections to the age at which Huntington's disease symptoms start to emerge. Dr. Yamamoto will talk about how Alfy may serve as a basis for a radically new treatment for Huntington's disease — and perhaps for other brain disorders.

Read our Newsletter to learn more about Ai’s work.