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Development of novel oligonucleotide delivery modalities for Huntington disease

Project Overview

Huntington disease (HD) is an inherited neurodegenerative disease that affects approximately 1 in 7500 individuals in the Western world. The cause of HD is a mutation in one copy of the HTT gene, which produces the toxic mutant huntingtin protein responsible for the brain cell loss associated with the disease. Targeting the root cause of HD by reducing mutant huntingtin in the brain should prevent or delay disease onset. One approach to reducing mutant huntingtin is to use specialized pieces of DNA, called antisense oligonucleotides (ASOs), to prevent production of the toxic mutant protein. Unfortunately, ASOs are unable to enter the brain when delivered in the blood and must be delivered directly into the fluid that bathes the brain, the cerebrospinal fluid or CSF. This procedure is invasive, and ASOs delivered this way do not reach all areas of the brain, including the area most affected by HD, the striatum. Therefore, we are developing new non-invasive strategies to enhance delivery of ASOs to the brain and body that we will test in HD mice and monkeys. We are also developing new ways to measure huntingtin in the brain so that we can evaluate changes in brain huntingtin in response to experimental therapies, like ASOs. We have previously shown that by quantifying mutant huntingtin in CSF, we are able to approximate levels of brain mutant huntingtin. We will continue development of methods to quantify CSF mutant huntingtin and develop complementary methods to measure the normal, non-mutant, huntingtin protein and other disease associated proteins. Finally, we will apply these methods to CSF collected from HD monkeys and patients over time, which will identify optimal methods for clinical assessment of changes in brain huntingtin. Together these studies will significantly advance huntingtin lowering therapies toward use in patients.

Principal Investigator

Blair Leavitt , University of British Columbia

Team Members

Danica Stanimirovic, National Research Council of Canada

Abedelnasser Abulrob, National Research Council of Canada

Jodi McBride, Oregon Health and Science University

Partners and Donors

Huntington Society of Canada

Project Ongoing

Development of novel oligonucleotide delivery modalities for Huntington disease

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    Other

  • Competition

    Brain Canada-Huntington Society of Canada Creating HD Clinician-Scientist-to-Patient Virtual Networks Multi-Investigator Research Initiative (MIRI)

  • Province

    British Columbia

  • Start Date

    2017

  • Total Grant Amount

    $950,000

  • Health Canada Contribution

    $475,000

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.

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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.

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    • 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
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