{"id":239342,"date":"2026-06-19T18:15:16","date_gmt":"2026-06-19T23:15:16","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2026\/06\/neuron-targeted-gene-therapy-rescues-multiple-phenotypes-of-stxbp1-related-disorders-in-mice-and-is-well-tolerated-in-nonhuman-primates"},"modified":"2026-06-19T18:15:16","modified_gmt":"2026-06-19T23:15:16","slug":"neuron-targeted-gene-therapy-rescues-multiple-phenotypes-of-stxbp1-related-disorders-in-mice-and-is-well-tolerated-in-nonhuman-primates","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2026\/06\/neuron-targeted-gene-therapy-rescues-multiple-phenotypes-of-stxbp1-related-disorders-in-mice-and-is-well-tolerated-in-nonhuman-primates","title":{"rendered":"Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates"},"content":{"rendered":"<p><\/p>\n<p><iframe style=\"display: block; margin: 0 auto; width: 100%; aspect-ratio: 4\/3; object-fit: contain;\" src=\"https:\/\/www.youtube.com\/embed\/PsRfxqRwUD0?feature=oembed\" frameborder=\"0\" allow=\"accelerometer; autoplay; encrypted-media; gyroscope;\n   picture-in-picture\" allowfullscreen><\/iframe><\/p>\n<p>Aeran and colleagues present research on targeted gene therapy vector engineering and pre-clinical testing of neuron-targeted AAV9-based constructs for <i>STXBP1<\/i>-related neurodevelopmental and epileptic encephalopathies. Candidate vectors designed to target specific neuronal types and detarget tissues associated with toxicity produced robust phenotypic reversal in <i>Stxbp1 <\/i>+\/\u2212 mice and were well tolerated in monkeys.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Aeran and colleagues present research on targeted gene therapy vector engineering and pre-clinical testing of neuron-targeted AAV9-based constructs for STXBP1-related neurodevelopmental and epileptic encephalopathies. Candidate vectors designed to target specific neuronal types and detarget tissues associated with toxicity produced robust phenotypic reversal in Stxbp1 +\/\u2212 mice and were well tolerated in monkeys.<\/p>\n","protected":false},"author":513,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,38,47],"tags":[],"class_list":["post-239342","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-engineering","category-neuroscience"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/239342","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/513"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=239342"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/239342\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=239342"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=239342"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=239342"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}