{"id":23096,"date":"2025-04-22T08:50:08","date_gmt":"2025-04-22T06:50:08","guid":{"rendered":"https:\/\/elainternational.eu\/?post_type=news_research&p=23096"},"modified":"2025-04-22T09:52:57","modified_gmt":"2025-04-22T07:52:57","slug":"evolving-the-aspa-transgene-for-a-next-generation-gene-therapy-for-canavan-disease","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/evolving-the-aspa-transgene-for-a-next-generation-gene-therapy-for-canavan-disease\/","title":{"rendered":"Evolving the ASPA transgene for a next generation gene therapy for Canavan Disease"},"content":{"rendered":"\n\n
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Evolving the ASPA transgene for a next generation gene therapy for Canavan Disease<\/strong><\/h2>\n <\/div>\n <\/div>\n<\/section>\n\n\n
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Lee Coffey, South East Technological University – Australia \/ ELA 2024-006I2<\/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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Canavan disease (CD) is a rare neurological disease, caused by mutation(s) in the ASPA gene, which encodes ASPA enzyme. These mutation(s) cause a deficiency in enzyme activity, resulting in excessive N-acetylaspartic acid (NAA) throughout the CNS. Excess NAA results in a breakdown of the myelin and white matter in the brain. Symptoms of CD include intellectual disability, loss of motor skills, abnormal muscle tone, visual degeneration and macrocephaly, with the majority of patients succumbing to the disease during childhood. Many treatments and therapies have been explored, with gene therapy emerging as the most promising, with much of this research conducted by Prof. Gao, Prof Gessler, and researchers at the Horae Gene therapy Centre, UMASS Chan Medical School.<\/p>\n

The project will focus on optimising the ASPA transgene, with a preliminary study completed, which has been observed to show disease rescue in the animal disease model. We aim to expand the study to identify an optimised ASPA transgene for therapeutic consideration; providing a high potency ASPA (via rAAV delivery), allowing for a vector dose reduction, improving the safety profile of the treatment, reducing potential adverse effects, reducing the cost of therapeutic production; all for a safe and efficacious Canavan gene therapy.<\/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":[120],"class_list":["post-23096","news_research","type-news_research","status-publish","hentry","news_research_categories-project","news_research_tags-canavan-disease"],"acf":[],"yoast_head":"\nEvolving the ASPA transgene for a next generation gene therapy for Canavan Disease - 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\/evolving-the-aspa-transgene-for-a-next-generation-gene-therapy-for-canavan-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Evolving the ASPA transgene for a next generation gene therapy for Canavan Disease - 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