Co-Investigator(s): Elena Torban (Research Institute of the McGill University Health Centre)
Cystinosis is a rare autosomal recessive disease caused by mutations of the CTNS gene. Without this gene, infants develop failure to thrive, progressive kidney failure and gradual deterioration of other organs.
Our project will show that a stabilized CTNS mRNA in a new lipid nanoparticle (LNP) forumlation can prevent deterioration of the kidneys, when delivered early in life to our Ctns-mutant mice and can reverse kidney damage if given later in life.
We will answer key questions about the design of therapeutic CTNS mRNAs and the novel LNP formulations that target the kidneys.