Project 462785

FAN1, a DNA repair nuclease, as a modifier of disease-associated repeat instability.

462785

FAN1, a DNA repair nuclease, as a modifier of disease-associated repeat instability.

$956,250
Project Information
Study Type: Unclear
Research Theme: Biomedical
Institution & Funding
Principal Investigator(s): Pearson, Christopher E
Institution: Hospital for Sick Children (Toronto)
CIHR Institute: Genetics
Program: Project Grant
Peer Review Committee: Genetics
Competition Year: 2022
Term: 5 yrs 0 mth
Abstract Summary

Huntington's disease (HD) is caused by mutations in repeated DNAs. If one considers a gene as though it were a sentence such as THE CAT ATE THE FAT RAT, typical mutations would be spelling errors, like THE GAT ATE THE FAT RAT. This mutation would be the same in all family members, all with the same symptoms. In repeat diseases the mutation would be THE CAT ATE THE FAT FAT FAT RAT, and their children would inherit a further mutation; THE CAT ATE THE FAT FAT FAT FAT FAT RAT and have the more severe disease than their parents. These mutations continue in affected tissues as the patient ages. Being able to arrest or reverse this repeat mutation presents a therapeutic pathway to reduce disease progression and severity. This proposal aims to characterize the role of FAN1 and its interaction with its partner protein as a modulator of HD progression and as a possible therapeutic target.

No special research characteristics identified

This project does not include any of the advanced research characteristics tracked in our database.

Keywords
Cag/Ctg Repeat Expansions Dna Repair Fan1 Nuclease Fan1-Mlh1 Interaction Huntington's Disease Mlh1 Complex Repeat Instability Spinocerebellar Ataxia (Scas) Transgenic Mice