{"id":2273,"date":"2023-07-18T19:22:17","date_gmt":"2023-07-18T23:22:17","guid":{"rendered":"https:\/\/cqdm.org\/en\/news-and-events\/sharp-screenable-assays-for-rna-binding-proteins-involved-in-brain-disorders\/"},"modified":"2024-09-25T16:25:56","modified_gmt":"2024-09-25T20:25:56","slug":"sharp-screenable-assays-for-rna-binding-proteins-involved-in-brain-disorders","status":"publish","type":"post","link":"https:\/\/cqdm.org\/en\/news-and-events\/sharp-screenable-assays-for-rna-binding-proteins-involved-in-brain-disorders\/","title":{"rendered":"ShARP: Screenable Assays for RNA-Binding Proteins Involved in Brain Disorders"},"content":{"rendered":"\n

Challenge:<\/strong> Brain\u00a0disorders such as autism, major depressive disorder and Alzheimer\u2019s disease are among the greatest medical challenges of our era. Many drugs targeting the central nervous system (CNS) have been developed to modulate G-protein coupled receptors, ion channels or transporters,\u00a0with mitigated success. There remains a shortage of disease-relevant targets that can be harnessed for drug discovery to treat the onset and progression of brain disorders\/diseases.<\/p>\n\n\n\n

Solution:<\/strong> This project focused\u00a0on RNA-binding proteins\u00a0(RBPs)\u00a0as untapped therapeutic targets because of their important role in the brain. Members of the\u00a0RBP\u00a0family provide powerful post-transcriptional control over cellular phenotype. The team\u2019s goal was to\u00a0develop a first-in-kind sensitive, adaptable, and validated\u00a0high\u2013throughput\u2013screening (HTS)\u00a0platform called\u00a0ShARP\u00a0for\u00a0Screenable\u00a0Assays for RNA-binding\u00a0Proteins, based\u00a0on the use of specific untranslated regions (UTRs) that are targeted by\u00a0RBPs\u00a0to modulate recruitment of messenger\u00a0RNA to ribosomes during protein synthesis.\u00a0Screening cell-based assays were developed for one\u00a0such\u00a0RBP,\u00a0FXR1, though not compatible with HTS\u00a0yet.\u00a0Novel 5\u2019 and 3\u2019-UTR reporter\u00a0libraries and associated RBP\u00a0sequence\u00a0databases were generated and\u00a0are available\u00a0for\u00a0RBP\u00a0screening.\u00a0The platform was tested in\u00a0partnership with Simon Fraser University\u00a0on FXR1,\u00a0a\u00a0demonstration\u00a0that\u00a0this RBP\u00a0could\u00a0be\u00a0targeted\u00a0and its activity inhibited or activated using small pharmacological molecules.\u00a0<\/p>\n\n\n\n

Achievements:<\/strong>\u00a0A first\u00a0platform\u00a0to screen\u00a0new\u00a0CNS-relevant\u00a0drugs that target the RBP family\u00a0was elaborated\u00a0and tested, opening\u00a0the path for RBPs\u00a0to be considered\u00a0and validated\u00a0as promising\u00a0therapeutic\u00a0targets\u00a0in neurological diseases.<\/p>\n\n\n\n

Principal Investigator:<\/strong>
Jean Martin Beaulieu
<\/strong>University of Toronto<\/td><\/tr>
Co-investigators<\/strong>
Keith Murai<\/strong>
McGill University
David Hunt<\/strong>
ImStar Therapeutics<\/td><\/tr>
Completed <\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong>Project<\/strong><\/td><\/tr>
$1,727,000 \/ 3 years<\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/strong><\/td><\/tr>
Supported by CQDM through:<\/strong>
– Merck
– Pfizer
– MEI<\/td><\/tr>
And by co-funding partners:\u00a0<\/strong>
– Brain Canada<\/td><\/tr><\/tbody><\/table><\/figure>\n","protected":false},"excerpt":{"rendered":"

Challenge: Brain\u00a0disorders such as autism, major depressive disorder and Alzheimer\u2019s disease are among the greatest medical challenges of our era. Many drugs targeting the central nervous system (CNS) have been developed to modulate G-protein coupled receptors, ion channels or transporters,\u00a0with mitigated success. There remains a shortage of disease-relevant targets that can be harnessed for drug…<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-2273","post","type-post","status-publish","format-standard","hentry","category-our-news"],"acf":[],"_links":{"self":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2273","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/comments?post=2273"}],"version-history":[{"count":1,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2273\/revisions"}],"predecessor-version":[{"id":4807,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/posts\/2273\/revisions\/4807"}],"wp:attachment":[{"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/media?parent=2273"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/categories?post=2273"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/cqdm.org\/en\/wp-json\/wp\/v2\/tags?post=2273"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}