{"id":30053,"date":"2024-12-20T12:03:12","date_gmt":"2024-12-20T17:03:12","guid":{"rendered":"https:\/\/genomequebec.wp.vortexdev.com\/?post_type=financed-project&#038;p=30053"},"modified":"2024-12-20T12:07:06","modified_gmt":"2024-12-20T17:07:06","slug":"an-mrna-strategy-to-restore-ctns-expression-in-cystinosis","status":"publish","type":"financed-project","link":"https:\/\/genomequebec.wp.vortexdev.com\/en\/funded-projects\/an-mrna-strategy-to-restore-ctns-expression-in-cystinosis\/","title":{"rendered":"An mRNA strategy to restore CTNS expression in Cystinosis"},"content":{"rendered":"\n<p><strong>Co-Investigator(s): <\/strong>Elena Torban (Research Institute of the McGill University Health Centre)<\/p>\n\n\n\n<p>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.<\/p>\n\n\n\n<p>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.<\/p>\n\n\n\n<p>We will answer key questions about the design of therapeutic CTNS mRNAs and the novel LNP formulations that target the kidneys.<\/p>\n","protected":false},"featured_media":0,"template":"","class_list":["post-30053","financed-project","type-financed-project","status-publish","hentry"],"acf":[],"_links":{"self":[{"href":"https:\/\/genomequebec.wp.vortexdev.com\/en\/wp-json\/wp\/v2\/financed-project\/30053","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/genomequebec.wp.vortexdev.com\/en\/wp-json\/wp\/v2\/financed-project"}],"about":[{"href":"https:\/\/genomequebec.wp.vortexdev.com\/en\/wp-json\/wp\/v2\/types\/financed-project"}],"wp:attachment":[{"href":"https:\/\/genomequebec.wp.vortexdev.com\/en\/wp-json\/wp\/v2\/media?parent=30053"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}