{"id":23612,"date":"2025-11-21T10:08:02","date_gmt":"2025-11-21T09:08:02","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&p=23612"},"modified":"2025-11-21T10:31:21","modified_gmt":"2025-11-21T09:31:21","slug":"deciphering-astrocyte-driven-neurotoxicity-in-x-ald-towards-targeted-neuroprotection-using-hdac3-inhibition","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/deciphering-astrocyte-driven-neurotoxicity-in-x-ald-towards-targeted-neuroprotection-using-hdac3-inhibition\/","title":{"rendered":"Deciphering astrocyte-driven neurotoxicity in X-ALD: towards targeted neuroprotection using HDAC3 inhibition"},"content":{"rendered":"\n\n
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Deciphering astrocyte-driven neurotoxicity in X-ALD: towards targeted neuroprotection using HDAC3 inhibition<\/h1>\n <\/div>\n <\/div>\n<\/section>\n\n\n
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Isabelle Weinhofer, Center for Brain Research, Medical University of Vienna \/ ELA 2025-034P4<\/em><\/p>\n <\/div>\n<\/section>\n\n\n\n\n\n

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Project abstract<\/strong><\/h5>\n <\/div>\n
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With a combined incidence of 1:14,700, X-linked adrenoleukodystrophy (X-ALD) is the most common monogenetically inherited leukodystrophy. It is caused by a genetic defect in the ABCD1 <\/em>gene that leads to the build-up of saturated very long-chain fatty acids (VLCFAs) in tissues and body fluids of patients. X-ALD shows a striking clinical heterogeneity with inflammatory cerebral ALD (CALD) being the most severe form. What triggers the onset of CALD is currently unknown, and the mechanisms resulting in the damage of brain cells and leading to the progressive loss of physical and mental functions remain unclear.<\/p>\n

In this project, we focus on a type of brain cells called astrocytes. Astrocytes normally support and protect nerve cells, but in X-ALD, the build-up of VLCFAs may cause a loss of helpful functions and instead contribute to brain damage. To address this, we will generate astrocytes and nerve cells from reprogrammed stem cells of patients with X-ALD and study their behaviour in cell culture. We will also test two different drugs, RGFP966 and RGFP109, that block an enzyme called HDAC3 in astrocytes. These drugs have shown promising results in studies with mice, and RGFP109 has already been safely tested in patients with the disorder Friedreich\u00b4s ataxia.<\/p>\n

Our goal is to selectively block HDAC3 using these compounds to prevent astrocytes from becoming harmful and to protect neurons from damage in patients with X-ALD. Together, our research could uncover new insights into how the lack of ABCD1 damages the brain and open up new possibilities for treatment.<\/p>\n <\/div>\n <\/div>\n<\/section>","protected":false},"excerpt":{"rendered":"","protected":false},"author":6,"featured_media":0,"parent":0,"template":"","news_research_categories":[127],"news_research_tags":[109],"class_list":["post-23612","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-x-linked-adrenoleukodystrophy"],"acf":[],"yoast_head":"\nDeciphering astrocyte-driven neurotoxicity in X-ALD: towards targeted neuroprotection using HDAC3 inhibition - ELA International<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/elainternational.eu\/en\/actualites_recherche\/deciphering-astrocyte-driven-neurotoxicity-in-x-ald-towards-targeted-neuroprotection-using-hdac3-inhibition\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Deciphering astrocyte-driven neurotoxicity in X-ALD: towards targeted neuroprotection using HDAC3 inhibition - 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