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Grant: Doctoral: Vanier Canada Graduate Scholarships

University of Alberta

$150,000.00

Towards treatment of Endothelial Corneal Dystrophies: Identification of correctors of mutant SLC4A11-misfolding

EF 2014-15

Résumé
The goal of my doctoral research is to find ways to cure and prevent three forms of genetic blindness (Congenital Hereditary Endothelial dystrophy, Fuchs Endothelial Corneal Dystrophy and Harboyan Syndrome). The cornea is the outermost layer of the eye. In corneal dystrophy diseases, the cornea fills with fluid that makes vision hazy and significantly impairs vision. At present, the only treatment for this condition is corneal transplant. The three conditions have in common that they are caused by mutations of a poorly understood gene called SLC4A11. The goal of our studies is to develop treatments for these corneal dystrophies, by testing the way to "fix" the diseased SLC4A11 protein in appropriate mice and cell culture models. In particular, I will screen for small molecules that can correct the defects in SLC4A11. These compounds hold promise to treat corneal blindness when applied as eye drops.
Programme
Doctoral: Vanier Canada Graduate Scholarships
Bénéficiaire
Emplacement
Alberta, Canada
Type de bénéficiaire
Academia
Type
Grant
Source
CIHR Funding Decisions Database

Tel que publié

Id
318315
Name
Malhotra Darpan
Region
Alberta
Country
Canada
Orgtype
Institution Paid; Research Institution
Instname2
Genetics
Theme2
Biomedical
Prcname2
Vanier Canada Graduate Scholarships CIHR
Projecttitle
Towards treatment of Endothelial Corneal Dystrophies: Identification of correctors of mutant SLC4A11-misfolding
Programtype2
Studentship Programs
Orgnameinp2
University of Alberta
Cihramount2
$150,000
Cihrequipment2
$0
Programname2
Doctoral: Vanier Canada Graduate Scholarships
Keyworddelim
Cornea; Endoplasmic Reticulum; Endothelial Corneal Dystrophy; Er Retained Proteins; High Throughput Assay; Molecular Chaperones; Protein Misfolding; Slc4a11; Small Molecule Correctors; Water Flux
Abstract
The goal of my doctoral research is to find ways to cure and prevent three forms of genetic blindness (Congenital Hereditary Endothelial dystrophy, Fuchs Endothelial Corneal Dystrophy and Harboyan Syndrome). The cornea is the outermost layer of the eye. In corneal dystrophy diseases, the cornea fills with fluid that makes vision hazy and significantly impairs vision. At present, the only treatment for this condition is corneal transplant. The three conditions have in common that they are caused by mutations of a poorly understood gene called SLC4A11. The goal of our studies is to develop treatments for these corneal dystrophies, by testing the way to "fix" the diseased SLC4A11 protein in appropriate mice and cell culture models. In particular, I will screen for small molecules that can correct the defects in SLC4A11. These compounds hold promise to treat corneal blindness when applied as eye drops.
Competitiondate
201411
Approvedterm2
3 yrs 0 mth
Pinamesdelim
Malhotra, Darpan
Approvedterm
3 ans 0 mois
Cihrcontribution2
~ $150,000~ $0~