{"id":23605,"date":"2025-11-21T09:41:02","date_gmt":"2025-11-21T08:41:02","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&p=23605"},"modified":"2025-11-21T10:16:12","modified_gmt":"2025-11-21T09:16:12","slug":"retargetingald-a-precision-hsc-gene-therapy-for-cerebral-x-ald","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/retargetingald-a-precision-hsc-gene-therapy-for-cerebral-x-ald\/","title":{"rendered":"ReTARGETingALD: A precision HSC gene therapy for cerebral X-ALD"},"content":{"rendered":"\n\n
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ReTARGETingALD: A precision HSC gene therapy for cerebral X-ALD<\/h1>\n <\/div>\n <\/div>\n<\/section>\n\n\n
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Alessandra Biffi, University of Padova – ELA 2025-002I2<\/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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This research project aims to develop a new treatment for X-linked adrenoleukodystrophy (X-ALD), a severe brain disorder caused by genetic mutations. Current treatments, while promising, have limitations. The proposed approach uses advanced gene editing techniques to insert a healthy copy of the ABCD1<\/em> gene into specific cells of the patient’s own blood stem cells.<\/p>\n

The researchers plan to use a method called CRISPR-Cas9 to precisely place the healthy gene in a way that could make the treatment more effective and safer than current gene therapy approaches. This technique is designed to produce long-lasting effects, while reducing the risks and limitations of currently available therapies.<\/p>\n

The team will test this new approach in laboratory models, including 3D structures that mimic human brain tissue and in mice with X-ALD. They will also compare it to a more traditional gene therapy method as a backup strategy.<\/p>\n

If successful, this innovative approach could overcome current limitations in X-ALD treatment and potentially serve as a model for treating other brain disorders. The project combines proven principles with cutting-edge technologies, aiming to translate scientific advancements into real-world medical treatments.<\/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-23605","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-x-linked-adrenoleukodystrophy"],"acf":[],"yoast_head":"\nReTARGETingALD: A precision HSC gene therapy for cerebral X-ALD - 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\/retargetingald-a-precision-hsc-gene-therapy-for-cerebral-x-ald\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"ReTARGETingALD: A precision HSC gene therapy for cerebral X-ALD - 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