Previously Funded Grants

Osama Al-Dalahmah, MD, DPhil
Institution: Columbia University Medical Center, NY
Project Title: (2019) The regional heterogeneity of Huntington’s disease pathology: Clues from diverse astrocytic responses
Project Title: (2020) Studying Huntington’s disease astrocytes in different parts of the brain: A regional study of the landscape of gene expression at the single cell level
2020 Nancy S. Wexler Young Investigator Prize

Cheryl Arrowsmith, PhD
Institution: University of Toronto, Ontario, Canada
Project Title: (2020) Unravelling the connections between the Huntington’s disease protein and our genetic material

Cheryl Arrowsmith, PhD with Rachel Harding, PhD
Institution: University of Toronto, Ontario, Canada
Project Title: (2021) Unravelling the connections between the Huntington’s disease protein and our genetic material

Anne Ast, PhD
Mentor: Erich E. Wanker, PhD
Institution: Max-Delbrück-Center for Molecular Medicine, Germany
Project Title: (2018) Manipulating seeding activity and proteotoxicity of amyloidogenic HTT aggregates by targeted amino acid exchanges

Natalia Moreira Barbosa, PhD
Mentor: Judith Frydman, PhD
Institution: Stanford University, Palo Alto, CA
Project Title: (2022) Linking Proteostasis and Mitochondrial dysfunction in Huntington’s Disease
2022 Nancy S. Wexler Young Investigator Prize

Costanza Ferrari Bardile, PhD
Mentor: Mahmoud Pouladi, PhD
Institution: University of British Columbia, Vancouver, Canada
Project Title: (2021) Understanding the role of oligodendroglia, brain cells that support neurons, in the pathology of Huntington’s disease

Kristina Becanovic, PhD
Institution: Karolinska Institutet, Sweden
Project Title: (2017) Towards the discovery of genetic modifiers and targets for future Huntington’s disease treatments

Abdellatif Benraiss, PhD
Institution: University of Rochester, NY
Project Title: (2020) White matter role in the pathology of Huntington’s disease

Marta Biagioli, PhD
Institution: University of Trento, Italy
Project Title: (2022) A circular RNA molecule to modify Huntington’s disease phenotypes

Veronica Ines Brito, PhD
Institution: University of Barcelona Instituto de Neurosciencias, Spain
Project Title: (2020) Exploring the role of RNA editing on the generation of pathogenic huntingtin fragments

Lauren Byrne, PhD
Mentor: Edward Wild, MD, PhD
Institution: University College London (UCL) Institute of Neurology, United Kingdom
Project Title: (2019) Advancing biofluid biomarkers for disease-modifying trials in Huntington’s disease

Mariacristina Capizzi
Institution: Paris Brain Institute, France
Project Title: (2022) Repairing the communication between brain cells in HD

Marie-Francoise Chesselet, MD, PhD
Institution: David Geffen School of Medicine at UCLA
Project Title: (2017) Preclinical evaluation of D-PUFAs as a therapeutic intervention for HD

Yoon H. Cho, PhD
Institution: University of Bordeaux, France
Project Title: (2017) Sleep as a window for a therapeutic clue against Huntington’s disease

Beverly Davidson, PhD with David Leib, PhD
Institution: Children’s Hospital of Philadelphia, PA
Project Title: (2021) New gene therapy delivery approaches for Huntington’s disease
2015 Leslie Gehry Prize for Innovation in Science

Amit Laxmikant Deshmukh, PhD
Mentor: Christopher E. Pearson, PhD
Institution: The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
Project Title: (2020) Role of FAN1 protein as modifier of the CAG repeat mutation in Huntington’s disease
Project Title: (2022) Role of FAN1 and MLH proteins as modifiers of the CAG repeat mutation in Huntington’s disease

Rivka Dikstein, PhD
Institution: The Weizmann Institute of Science, Rehovot, Israel
Project Title: (2019) Unraveling the role of Spt4/Spt5 in inherited neurodegenerative diseases using newly discovered pharmacological tools

Hassan Fakih, PhD
Mentor: Anastasia Khvorova, PhD
Institution: University of Massachusetts Chan Medical School, Worcester, MA
Project Title: (2022) Developing nucleic acid nanoparticles for improved RNAi therapeutics to treat Huntington’s disease

Steven Finkbeiner, MD, PhD with Leslie Thompson, PhD
Institution: The J. David Gladstone Institutes; University of California, San Francisco
Project Title: (2017) Assessment of WGS-derived genetic modifiers in differentiated HD-derived iPSCs
2013 Leslie Gehry Prize for Innovation in Science
2022 Leslie Gehry Prize for Innovation in Science

Brent Fitzwalter, PhD
Mentor: Myriam Heiman, PhD
Institution: Broad Institute of Massachusetts Institute of Technology and Harvard University
Project Title: (2019) Neuroprotection in Huntington’s Disease

Terence Gall-Duncan, PhD
Mentor: Christopher E. Pearson, PhD
Institution: The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada
Project Title: (2022) Reversing the expansion mutation that causes Huntington’s disease

Michelle Gray, PhD with Marissa Dean, MD and Steven Pogwizd, MD
Institution: University of Alabama at Birmingham
Project Title: (2021) Heart problems in Huntington’s disease

Carolina Gubert, PhD
Mentor: Anthony Hannan, PhD
Institution: The Florey Institute of Neuroscience and Mental Health, University of Melbourne, Australia
Project Title: (2021) Targeting the gut bugs in Huntington’s disease: Identifying novel therapeutic opportunities

Rachel Harding, PhD with Cheryl Arrowsmith, PhD
Institution: University of Toronto, Ontario, Canada
Project Title: (2021) Unravelling the connections between the Huntington’s disease protein and our genetic material
Project Title: (2023) Structural Investigation of Huntingtin-RNA Interactions

Sarah Hernandez, PhD
Mentor: Leslie Thompson, PhD
Institution: University of California, Irvine
Project Title: (2021) Understanding how connections between cells can affect permeability of the brain in HD
2021 Nancy S. Wexler Young Investigator Prize

Richard A. Hickman, MD
Institution: Columbia University Medical Center, NY
Project Title: (2019) Generation of Striatal Neurons from HD patient-derived fibroblasts: a feasibility study with direct correlation to human neuropathology

Richard A. Hickman, MD with Jean Paul Vonsattel, MD
Institution: New York Brain Bank, Columbia University Irving Medical Center, NY
Project Title: (2020) Human tissue banking for the Huntington’s disease research community

Ben Hoffstrom, PhD with Ai Yamamoto, PhD, Columbia University
Institution: Fred Hutchinson Cancer Research Center, Seattle, WA
Project Title: (2017) Creating a monoclonal antibody against Alfy
2020 Leslie Gehry Prize for Innovation in Science (Yamamoto)

Ali Khoshnan, PhD
Institution: California Institute of Technology, Pasadena
Project Title: (2019) Editing of gut bacteria to reduce brain pathology in Huntington’s disease
Project Title: (2020) Developing gut-based therapies for HD

Hyeseung Lee, PhD
Mentor: Myriam Heiman, PhD
Institution: Picower Institute for Learning and Memory, Massachusetts Institute of Technology, Cambridge
Project Title: (2021) Therapeutic targeting of Huntington’s disease with key regulators of innate immunity

David Leib, PhD with Beverly Davidson, PhD
Institution: Children’s Hospital of Philadelphia, University of Pennsylvania
Project Title: (2021) New gene therapy delivery approaches for Huntington’s disease
2015 Leslie Gehry Prize for Innovation in Science (Davidson)

Pan Li, PhD
Mentor: Russell Margolis, PhD
Institution: Johns Hopkins University, Baltimore, MD
Project Title: (2017) A high throughput screen for small molecules enhancing the expression of HTTAS-a novel therapeutic strategy for HD

Xueyi Li, PhD
Institution: Massachusetts General Hospital, Harvard Medical School, Boston, MA
Project Title: (2021) Mechanism of white matter loss in Huntington’s disease

Ryan Lim, PhD
Institution: University of California, Irvine
Project Title: (2020) Interactions between metabolism, gene expression, and gender in Huntington’s disease

Boxun Lu
Institution: Fudan University, Shanghai, China
Project Title: (2017) Validation of a Gpr52 antagonist’s effect on mHTT levels and toxicity

James Mackay, PhD
Mentor: Lynn A. Raymond, MD, PhD, FRCPC
Institution: University of British Columbia, Vancouver, Canada
Project Title: (2021) Assessing early changes in sensory-related brain activity in Huntington’s disease

Srivathsa Magadi, PhD
Mentor: Walker Jackson, PhD
Institution: Linkoping University, Sweden
Project Title: (2022) Modulate immune cells of the brain to alleviate Huntington’s disease

Roy Maimon, PhD
Mentor: Don W. Cleveland, PhD
Institution: University of California, San Diego
Project Title: (2020) Therapy development for Huntington’s disease using in vivo conversion of astrocytes into striatal neurons

Alejandro Mas-Monteys, PhD
Institution: Children’s Hospital of Philadelphia, University of Pennsylvania
Project Title: (2018) Novel approaches for knock down control

Zachariah L. McLean, PhD
Mentor: James F. Gusella, PhD
Institution: Massachusetts General Hospital, Harvard Medical School, Boston, MA
Project Title: (2021) Identifying modifiers of Huntington’s disease CAG repeat expansion and its consequent disruption of messenger RNA

Jenny Morton, PhD, SCD, FRSB
Institution: University of Cambridge, United Kingdom
Project Title: (2020) Can core body temperature be used as a readout for changes in metabolism in Huntington’s disease?

Jose F. Moruno-Manchon, PhD
Mentor: Andrey S. Tsvetkov, PhD
Institution: The University of Texas McGovern Medical School, Houston
Project Title: (2017) Sphingosine kinase 2 and DNA damage in neurons – A lipid kinase, DNA damage and Huntington’s disease

Christian Neri, PhD
Institution: INSERM, Paris, France
Project Title: (2017) Modeling the role of extracellular vesicles in HD pathogenesis

Christopher Ng, PhD
Mentor: David Housman, PhD
Institution: Massachusetts Institute of Technology, Cambridge
Project Title: (2017) Characterization of genetic variants that modify age of onset in Huntington’s disease

Daniel O’Reilly, PhD
Mentor: Anastasia Khvorova, PhD
Institution: University of Massachusetts Chan Medical School, Worcester, MA
Project Title: (2020) Understanding the role of aggregates in Huntington’s disease

Izabella A. Pena, PhD
Mentor: Myriam Heiman, PhD
Institution: Picower Institute for Learning and Memory, Massachusetts Institute of Technology, Cambridge
Project Title: (2021) Using metabolism to measure defects in cellular self-cleaning in Huntington’s disease
Project Title: (2022) Investigating mitochondrial defects in the most vulnerable neurons in Huntington’s disease

Ellen Penney, MD, PhD
Institution: Massachusetts General Hospital, Harvard Medical School, Boston, MA
Project Title: (2018) R-loops in HD: Somatic Expansion and DNA Repair

Ashutosh Phadte, PhD
Mentor: Anna Pluciennik, PhD
Institution: Thomas Jefferson University, Philadelphia, PA
Project Title: (2021) Oxidative damage and CAG expansions in Huntington’s disease

Anna Pluciennik, PhD
Institution: Thomas Jefferson University, Philadelphia, PA
Project Title: (2018) Crosstalk between DNA repair pathways in Huntington’s disease

Paul Ranum, PhD
Mentor: Beverly Davidson, PhD
Institution: Children’s Hospital of Philadelphia, University of Pennsylvania
Project Title: (2019) High throughput quantification of gene expression and mRNA structure from single-cells in the HD brain

Piere Rodriguez-Aliaga, PhD
Mentor: Judith Frydman, PhD
Institution: Stanford University, Palo Alto, CA
Project Title: (2018) Dissection of the functional and structural communication within the Huntingtin protein and its interaction with the human TRiC chaperonin
Project Title: (2019) Structural differences between the pathogenic and non-pathogenic Huntingtin: a single- molecule approach
Project Title: (2021) How disease-causing mutations in the Huntingtin protein render it toxic

Jennie C. L. Roy, PhD
Mentor: Ricardo Mouro Pinto, PhD
Institution: Massachusetts General Hospital, Harvard Medical School, Boston, MA
Project Title: (2020) Testing of novel drugs targeting CAG repeat expansions as candidate therapeutics for Huntington’s disease

Ghazaleh  Sadri-Vakili, PhD
Institution: Massachusetts General Hospital, Harvard Medical School, Boston, MA
Project Title: (2017) Alterations in Hippo/YAP signaling as a pathogenic mechanism in Huntington’s disease

Partha Sarkar, PhD
Institution: University of Texas Medical Branch at Galveston
Project Title: (2021) Mechanism of mitochondrial dysfunction in Huntington’s disease
Project Title: (2023) Loss of Genome Integrity in Huntington’s Disease

Matthew Scaglione, PhD
Institution: Medical College of Wisconsin and Duke University School of Medicine
Project Title: (2018) Analysis of a novel class of molecular chaperones that suppress polyglutamine aggregation

Chiara Scaramuzzino, PhD
Institution: Grenoble Institut des Neurosciences-INSERM, France
Project Title: (2021) Impact of mutant huntingtin on retrograde axonal routing of survival signals

Sridhar Selvaraj, PhD
Mentor: Matthew Porteus, MD, PhD
Institution: Stanford University, Palo Alto, CA
Project Title: (2022) Development of a stem-cell based therapy for Huntington’s disease

Anne Simonsen, PhD
Institution: University of Oslo, Norway
Project Title: (2017) Alfy at the Battlefront against Huntingtons Disease

Charlene Smith-Geater, PhD
Mentor: Leslie M. Thompson, PhD
Institution: University of California, Irvine
Project Title: (2017) PIAS1 network in HD induced pluripotent stem cells – Dressing Huntington: The mistake in the protein that causes Huntington’s disease can be very subtle!

Sophie St-Cyr, PhD
Mentor: Beverly L. Davidson, PhD
Institution: Children’s Hospital of Philadelphia, University of Pennsylvania
Project Title: (2021) Understanding the underlying mechanism of the heart pathology in Huntington’s disease

Joan Steffan, PhD
Institution: University of California, Irvine
Project Title: (2017) Analysis of an ubiquitin-binding domain within Huntingtin – Analysis of how the Huntingtin protein helps clean up cellular trash
Project Title: (2020) Identification of Huntingtin-dependent cellular trash collection pathways

Andrew F. Teich, MD, PhD
Institution: New York Brain Bank, Columbia University Irving Medical Center, NY
Project Title: (2021) Human tissue banking for the Huntington’s disease research community

Leslie Thompson, PhD with Steve Finkbeiner, MD, PhD
Institution: University of California, Irvine
Project Title: (2017) Assessment of WGS-derived genetic modifiers in differentiated HD-derived iPSCs
2013 Leslie Gehry Prize for Innovation in Science (Thompson)
2022 Leslie Gehry Prize for Innovation in Science (Finkbeiner)

Nicholas Todd, PhD
Institution: Brigham and Women’s Hospital, Harvard Medical School, Boston, MA
Project Title: (2019) Targeted Delivery of HD Gene Therapeutics
Project Title: (2020) Improving delivery of Huntington’s disease therapies to the brain Project Title: (2021) Delivery of Huntington’s disease gene therapeutics to the brain

Ray Truant, PhD
Institution: McMaster University, Hamilton, Ontario, Canada
Project Title: (2019) DNA Damage Repair Links to Energy Metabolism Defects in HD: Defining New Therapeutic Targets
Project Title: (2022) Imaging huntingtin at DNA sites at the atomic level

Jean Paul Vonsattel, MD with Richard A. Hickman, MD
Institution: New York Brain Bank, Columbia University Irving Medical Center, NY
Project Title: (2020) Human tissue banking for the Huntington’s disease research community

Mary Wertz, PhD
Mentor: Myriam Heiman, PhD
Institution: Broad Institute of Massachusetts Institute of Technology and Harvard University
Project Title: (2017) In vivo genome-wide genetic screening for modifiers of mutant Huntingtin toxicity

Chao Wu, PhD
Mentor: Guo-Min Li, PhD
Institution: University of Texas Southwestern Medical Center, Dallas
Project Title: (2020) Mutant huntingtin promotes CAG repeat expansion

Gong-Her Wu, PhD
Mentor: Wah Chiu, PhD
Institution: Stanford University, Palo Alto, CA
Project Title: (2019) Deciphering mutated huntingtin aggregates and cellular architecture in Huntington’s disease neuron by cryogenic electron microscopy
Project Title: (2021) Using cryogenic electron microscopy to visualize protein aggregates and subcellular component structures in Huntington’s disease neurons

Ai Yamamoto, PhD
Institution: Columbia University Medical Center, NY
Project Title: (2017) ALFY-mediated Degradation and HD
Project Title: (2020) Determining how the protein Alfy may improve Huntington’s disease-like symptoms
2020 Leslie Gehry Prize for Innovation in Science

William Yang, MD, PhD
Institution: David Geffen School of Medicine at UCLA j
Project Title: (2017) Novel Fan1 Knockin Mice to Facilitate Huntington’s Disease Genetic Modifier Research
Project Title: (2019) Novel FAN1 Knock-in Mice to Study the Role of FAN1 in Normal Brain Function and HD Pathogenesis
2014 Leslie Gehry Prize for Innovation in Science

Andrew Yoo, PhD
Institution: Washington University School of Medicine, St. Louis, MO
Project Title: (2017) Modeling Huntington’s Disease with Patient Neurons Generated by Direct Neuronal Reprogramming
Project Title: (2018) Testing the Role of Genetic Modifiers of Huntington’s Disease with Directly Reprogrammed Patient Neurons
Project Title: (2021) Increasing resilience against neurodegeneration in Huntington’s disease patient-derived neurons

Michael Zody, PhD
Institution: New York Genome Center, NY
Project Title: (2020) New York Genome Center Huntington’s Disease Brain Genotyping Project

Detailed descriptions for currently funded research projects.

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