Skip to content
Project Directory
  • Français
Donate Now
  • Français
  • About
    • What We Do
    • EDI Action Plan
    • Leadership
    • Team
    • Annual Report
    • Publications
    • Careers
  • Brain Conditions
    • One Brain
    • ALS
    • Autism (ASD)
    • Brain Cancer
    • Brain Injury
    • Dementia
    • Epilepsy
    • Mental Illness
    • Multiple Sclerosis
    • Parkinson’s
    • Stroke
    • More
  • Research
    • Programs
    • Funding Opportunities
    • Program Partners
    • Announcements
  • Impact
    • Research Impact Stories
    • Equity, Diversity and Inclusion
    • Brain Health in Indigenous Communities
    • Women’s Brain Health
    • Mind Over Matter
  • How You Can Help
    • Ways to Give
    • Start a Fundraiser
    • Workplace Giving
    • The Great Minds

Funded Grants

Back to results

Modulating the ALS-associated C9orf72 repeat expansion in a murine model for therapeutic benefit

Project Overview

Mutations in a gene called C9ORF72 are the most common genetic cause of ALS. These mutations are unique in that unlike most other ALS-linked genes, where there is often a mistake in a single piece of DNA, C9ORF72 mutations involve a section of DNA that is abnormally repeated hundreds or even thousands of times. People with these repeat mutations make less of the normal C9ORF72 protein, but also produce more toxic substances in their motor neurons.

Dr. Christopher Pearson is an expert in studying repeat DNA expansions in disease and through his recent work, he has identified a previously unobserved characteristic about the C9ORF72 expansion mutation that makes it a potentially druggable target.

In this study, Dr. Pearson will determine if treating C9ORF72 model mice with a compound called fluorodeoxyuridine (FUdR) can cause these repeated DNA sections to shrink back to a less toxic size and restore production of the normal C9ORF72 protein. In collaboration with Dr. Ekaterina Rogaeva, an expert in studying a process called DNA called methylation, which can also alter C9ORF72 production, the team will examine how this process is affected by FUdR treatment.

The results gained from this project will help researchers to better understand the unique mutations in C9ORF72 and provide insight into novel therapeutic avenues to treat this form of the disease. If successful, the researchers would embark on a larger study to confirm the potential for FUdR as a treatment for C9ORF72-linked ALS.

Principal Investigator

Christopher Pearson , The Hospital for Sick Children

Team Members

Ekaterina Rogaeva, University of Toronto

Partners and Donors

ALS Canada

Project Complete

Modulating the ALS-associated C9orf72 repeat expansion in a murine model for therapeutic benefit

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Ontario

  • Start Date

    2021

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Contact Us

1200 McGill College Avenue
Suite 1600, Montreal, Quebec
H3B 4G7

+1 (514) 989-2989 info@braincanada.ca

Please note all online donations will receive an electronic tax receipt, issued by Brain Canada Foundation.

Our Donors

Playing with Marbles Podcast

Join us and take a journey to the real last great frontier – the brain.

Listen

Subscribe to Brain News

Receive our monthly electronic newsletter with updates on funded projects, upcoming events and breakthroughs in brain research.

Sign Up

Territorial acknowledgement

The offices of Brain Canada Foundation are located on the traditional, ancestral territory of the Kanien'kehá:ka Peoples, a place which has long served as a site of meeting and exchange amongst nations. We honour and pay respect to elders past, present and emerging, and dedicate ourselves to moving forward in the spirit of partnership, collaboration, and reconciliation. In our work, we focus our efforts on the Truth and Reconciliation Commission’s Calls to Action, particularly those that pertain to improving health for Indigenous Peoples and that focus on advancing our own learning on Indigenous issues.

© 2025 Brain Canada Foundation

Registration number: 89105 2094 RR0001

  • Terms and Conditions
  • Privacy Policy

Design by Field Trip & Co

  • About
    • What We Do
    • EDI Action Plan
    • Leadership
    • Team
    • Annual Report
    • Publications
    • Careers
  • Brain Conditions
    • One Brain
    • ALS
    • Autism (ASD)
    • Brain Cancer
    • Brain Injury
    • Dementia
    • Epilepsy
    • Mental Illness
    • Multiple Sclerosis
    • Parkinson’s
    • Stroke
    • More
  • Research
    • Programs
    • Funding Opportunities
    • Program Partners
    • Announcements
  • Impact
    • Research Impact Stories
    • Equity, Diversity and Inclusion
    • Brain Health in Indigenous Communities
    • Women’s Brain Health
    • Mind Over Matter
  • How You Can Help
    • Ways to Give
    • Start a Fundraiser
    • Workplace Giving
    • The Great Minds
Project Directory
Donate Now