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Novel Gene Therapy Approaches for Targeting Aging in MND

  • Shadfar, Sina (Primary Chief Investigator)
  • Atkin, Julie (Chief Investigator)
  • Dhungel, Bijay (Chief Investigator)
  • Thordarson, Pall (Partner Investigator)

Project: Research

Project Details

Description

FightMND IMPACT Grant
Our research aims to understand how aging contributes to motor neuron disease (MND) and explore novel treatments. Aging increases DNA damage, a major factor in aging-related diseases. Sirtuins (SIRTs), proteins linked to aging, show promise in extending lifespan and preventing DNA damage. SIRT7, one of these proteins, has unique properties suggesting it could be particularly effective in MND. Despite some research on other SIRTs in MND, SIRT7's role remains largely unexplored. Our hypothesis is that SIRT7 is reduced in MND, and boosting it could protect against DNA damage and neurodegeneration. We plan to investigate how SIRT7 works in MND and test if delivering it via gene therapy can protect against MND in mice with TDP-43 pathology, a common feature of the disease. Our study will provide insights into SIRT7's potential as a therapeutic target for MND and lay the groundwork for future research in this area.
AcronymFightMND 24
StatusActive
Effective start/end date3/03/252/03/27