{"id":4268,"date":"2025-01-07T04:29:00","date_gmt":"2025-01-07T04:29:00","guid":{"rendered":"http:\/\/biogroupvietnam-worldpress.local\/2025\/08\/18\/gene-and-cell-therapies-take-aim-at-parkinsons-disease\/"},"modified":"2025-09-29T14:00:14","modified_gmt":"2025-09-29T07:00:14","slug":"lieu-phap-gen-va-te-bao-huong-den-benh-parkinson","status":"publish","type":"post","link":"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/","title":{"rendered":"Li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson"},"content":{"rendered":"<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson l\u00e0 c\u00e1c ph\u01b0\u01a1ng ph\u00e1p m\u1edbi \u00edt x\u00e2m l\u1ea5n nh\u01b0ng l\u00e2u d\u00e0i h\u1ee9a h\u1eb9n s\u1ebd ng\u0103n ch\u1eb7n v\u00e0 th\u1eadm ch\u00ed \u0111\u1ea3o ng\u01b0\u1ee3c qu\u00e1 tr\u00ecnh tho\u00e1i h\u00f3a th\u1ea7n kinh.<\/span><\/p>\n<p>&nbsp;<\/p>\n<figure class=\"image\"><img fetchpriority=\"high\" decoding=\"async\" style=\"aspect-ratio:2240\/1260\" src=\"https:\/\/biogroupvietnam.com\/wp-content\/uploads\/2025\/09\/lieu-phap-gen-te-bao-goc-dieu-tri-parkinson.png\" alt=\"Ph\u1ee5c h\u1ed3i ho\u1eb7c t\u00e1i t\u1ea1o t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ecb t\u1ed5n th\u01b0\u01a1ng l\u00e0 \u01b0u \u0111i\u1ec3m c\u1ee7a li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson&nbsp;\" width=\"2240\" height=\"1260\"><figcaption><span style=\"font-family:Arial, Arial_EmbeddedFont, Arial_MSFontService, sans-serif;font-size:12pt\"><i>Ph\u1ee5c h\u1ed3i ho\u1eb7c t\u00e1i t\u1ea1o t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ecb t\u1ed5n th\u01b0\u01a1ng l\u00e0 \u01b0u \u0111i\u1ec3m c\u1ee7a li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson<\/i>&nbsp;<\/span><\/figcaption><\/figure>\n<div id=\"ez-toc-container\" class=\"ez-toc-v2_0_80 counter-hierarchy ez-toc-counter ez-toc-grey ez-toc-container-direction\">\n<div class=\"ez-toc-title-container\">\n<p class=\"ez-toc-title\" style=\"cursor:inherit\">M\u1ee5c l\u1ee5c<\/p>\n<span class=\"ez-toc-title-toggle\"><a href=\"#\" class=\"ez-toc-pull-right ez-toc-btn ez-toc-btn-xs ez-toc-btn-default ez-toc-toggle\" aria-label=\"Toggle Table of Content\"><span class=\"ez-toc-js-icon-con\"><span class=\"\"><span class=\"eztoc-hide\" style=\"display:none;\">Toggle<\/span><span class=\"ez-toc-icon-toggle-span\"><svg style=\"fill: #999;color:#999\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" class=\"list-377408\" width=\"20px\" height=\"20px\" viewBox=\"0 0 24 24\" fill=\"none\"><path d=\"M6 6H4v2h2V6zm14 0H8v2h12V6zM4 11h2v2H4v-2zm16 0H8v2h12v-2zM4 16h2v2H4v-2zm16 0H8v2h12v-2z\" fill=\"currentColor\"><\/path><\/svg><svg style=\"fill: #999;color:#999\" class=\"arrow-unsorted-368013\" xmlns=\"http:\/\/www.w3.org\/2000\/svg\" width=\"10px\" height=\"10px\" viewBox=\"0 0 24 24\" version=\"1.2\" baseProfile=\"tiny\"><path d=\"M18.2 9.3l-6.2-6.3-6.2 6.3c-.2.2-.3.4-.3.7s.1.5.3.7c.2.2.4.3.7.3h11c.3 0 .5-.1.7-.3.2-.2.3-.5.3-.7s-.1-.5-.3-.7zM5.8 14.7l6.2 6.3 6.2-6.3c.2-.2.3-.5.3-.7s-.1-.5-.3-.7c-.2-.2-.4-.3-.7-.3h-11c-.3 0-.5.1-.7.3-.2.2-.3.5-.3.7s.1.5.3.7z\"\/><\/svg><\/span><\/span><\/span><\/a><\/span><\/div>\n<nav><ul class='ez-toc-list ez-toc-list-level-1 ' ><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-1\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Cac_lieu_phap_dieu_tri_Parkinson_pho_bien\" >C\u00e1c li\u1ec7u ph\u00e1p \u0111i\u1ec1u tr\u1ecb Parkinson ph\u1ed5 bi\u1ebfn<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-2\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Lieu_phap_te_bao_goc_dieu_tri_Parkinson\" >Li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-3\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Lieu_phap_gen_dieu_tri_Parkinson_%E2%80%93_Kich_thich_su_tai_sinh_cua_te_bao_than_kinh\" >Li\u1ec7u ph\u00e1p gen \u0111i\u1ec1u tr\u1ecb Parkinson &#8211; K\u00edch th\u00edch s\u1ef1 t\u00e1i sinh c\u1ee7a t\u1ebf b\u00e0o th\u1ea7n kinh<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-4\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Giam_su_hoat_dong_qua_muc_cua_cac_te_bao_than_kinh_van_dong\" >Gi\u1ea3m s\u1ef1 ho\u1ea1t \u0111\u1ed9ng qu\u00e1 m\u1ee9c c\u1ee7a c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh v\u1eadn \u0111\u1ed9ng<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-5\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Lieu_phap_gen_dieu_tri_parkinson_khong_can_phau_thuat\" >Li\u1ec7u ph\u00e1p gen \u0111i\u1ec1u tr\u1ecb parkinson kh\u00f4ng c\u1ea7n ph\u1eabu thu\u1eadt<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-6\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Giam_tich_tu_Protein_%CE%B1-Synuclein\" >Gi\u1ea3m t\u00edch t\u1ee5 Protein \u03b1-Synuclein<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-7\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Tuong_lai_dieu_tri_Parkinson\" >T\u01b0\u01a1ng lai \u0111i\u1ec1u tr\u1ecb Parkinson<\/a><\/li><li class='ez-toc-page-1 ez-toc-heading-level-2'><a class=\"ez-toc-link ez-toc-heading-8\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/#Tai_lieu_tham_khao\" >T\u00e0i li\u1ec7u tham kh\u1ea3o<\/a><\/li><\/ul><\/nav><\/div>\n<h2><span class=\"ez-toc-section\" id=\"Cac_lieu_phap_dieu_tri_Parkinson_pho_bien\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>C\u00e1c li\u1ec7u ph\u00e1p \u0111i\u1ec1u tr\u1ecb Parkinson ph\u1ed5 bi\u1ebfn<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Parkinson l\u00e0 m\u1ed9t b\u1ec7nh l\u00fd tho\u00e1i h\u00f3a th\u1ea7n kinh, \u0111\u1eb7c tr\u01b0ng b\u1edfi s\u1ef1 m\u1ea5t m\u00e1t c\u1ee7a c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh s\u1ea3n xu\u1ea5t dopamine trong n\u00e3o. Ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb ph\u1ed5 bi\u1ebfn nh\u1ea5t l\u00e0 s\u1eed d\u1ee5ng&nbsp;<strong>levodopa<\/strong>, m\u1ed9t ti\u1ec1n ch\u1ea5t dopamine c\u00f3 kh\u1ea3 n\u0103ng th\u00e2m nh\u1eadp v\u00e0o n\u00e3o \u0111\u1ec3 c\u1ea3i thi\u1ec7n tri\u1ec7u ch\u1ee9ng. Tuy nhi\u00ean, levodopa ch\u1ec9 l\u00e0 gi\u1ea3i ph\u00e1p t\u1ea1m th\u1eddi, khi t\u1ebf b\u00e0o th\u1ea7n kinh ti\u1ebfp t\u1ee5c ch\u1ebft \u0111i, thu\u1ed1c d\u1ea7n m\u1ea5t t\u00e1c d\u1ee5ng. M\u1ed9t s\u1ed1 b\u1ec7nh nh\u00e2n \u0111\u01b0\u1ee3c ch\u1ec9 \u0111\u1ecbnh ph\u1eabu thu\u1eadt&nbsp;<strong>k\u00edch th\u00edch n\u00e3o s\u00e2u<\/strong> (Deep Brain Stimulation &#8211; DBS) \u0111\u1ec3 \u0111i\u1ec1u ch\u1ec9nh ho\u1ea1t \u0111\u1ed9ng b\u1ea5t th\u01b0\u1eddng c\u1ee7a n\u00e3o, nh\u01b0ng ph\u01b0\u01a1ng ph\u00e1p n\u00e0y ti\u1ec1m \u1ea9n r\u1ee7i ro nh\u01b0 nhi\u1ec5m tr\u00f9ng, xu\u1ea5t huy\u1ebft v\u00e0 \u0111\u1ed9t qu\u1ef5.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">M\u1eb7c d\u00f9 c\u00e1c ph\u01b0\u01a1ng ph\u00e1p tr\u00ean h\u1ed7 tr\u1ee3 c\u1ea3i thi\u1ec7n ch\u1ea5t l\u01b0\u1ee3ng s\u1ed1ng cho b\u1ec7nh nh\u00e2n, nh\u01b0ng kh\u00f4ng gi\u1ea3i quy\u1ebft \u0111\u01b0\u1ee3c g\u1ed1c r\u1ec5 v\u1ea5n \u0111\u1ec1 nh\u01b0 ph\u1ee5c h\u1ed3i ho\u1eb7c t\u00e1i t\u1ea1o t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ecb t\u1ed5n th\u01b0\u01a1ng. \u0110\u00e2y l\u00e0 l\u00fac c\u00e1c gi\u1ea3i m\u1edbi<strong>&nbsp;<\/strong>\u00edt x\u00e2m l\u1ea5n h\u01a1n \u0111\u01b0\u1ee3c ch\u00fa tr\u1ecdng ph\u00e1t tri\u1ec3n.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Lieu_phap_te_bao_goc_dieu_tri_Parkinson\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>Li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><a target=\"_blank\" rel=\"noopener noreferrer\" href=\"https:\/\/aspenneuroscience.com\/\"><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\">Aspen Neuroscience&nbsp;<\/span><\/a><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">\u0111ang ti\u00ean phong trong vi\u1ec7c ph\u00e1t tri\u1ec3n&nbsp;<strong>li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c t\u1ef1 th\u00e2n<\/strong> \u0111\u1ec3 thay th\u1ebf v\u00e0 thi\u1ebft l\u1eadp l\u1ea1i c\u00e1c k\u1ebft n\u1ed1i c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ecb m\u1ea5t. \u0110\u1eb7c \u0111i\u1ec3m n\u1ed5i b\u1eadt c\u1ee7a ph\u01b0\u01a1ng ph\u00e1p n\u00e0y l\u00e0 s\u1eed d\u1ee5ng t\u1ebf b\u00e0o c\u1ee7a ch\u00ednh b\u1ec7nh nh\u00e2n, gi\u00fap lo\u1ea1i b\u1ecf nguy c\u01a1 \u0111\u00e0o th\u1ea3i mi\u1ec5n d\u1ecbch.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Quy tr\u00ecnh b\u1eaft \u0111\u1ea7u t\u1eeb vi\u1ec7c l\u1ea5y m\u1eabu t\u1ebf b\u00e0o da c\u1ee7a b\u1ec7nh nh\u00e2n, sau \u0111\u00f3 chuy\u1ec3n \u0111\u1ed5i ch\u00fang th\u00e0nh t\u1ebf b\u00e0o g\u1ed1c \u0111a n\u0103ng c\u1ea3m \u1ee9ng (iPSCs) v\u00e0 bi\u1ec7t h\u00f3a th\u00e0nh ti\u1ec1n th\u00e2n t\u1ebf b\u00e0o th\u1ea7n kinh dopaminergic. Nh\u1eefng t\u1ebf b\u00e0o n\u00e0y kh\u00f4ng ch\u1ec9 tr\u1ebb h\u01a1n v\u1ec1 \u0111\u1ed9 d\u00e0i telomere m\u00e0 c\u00f2n c\u1ea3i thi\u1ec7n kh\u1ea3 n\u0103ng ho\u1ea1t \u0111\u1ed9ng c\u1ee7a ty th\u1ec3. Sau khi \u0111\u01b0\u1ee3c c\u1ea5y gh\u00e9p, ch\u00fang c\u00f3 kh\u1ea3 n\u0103ng t\u00e1i c\u1ea5u tr\u00fac m\u1ea1ng l\u01b0\u1edbi th\u1ea7n kinh v\u00e0 s\u1ea3n xu\u1ea5t dopamine c\u1ea7n thi\u1ebft.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson \u0111\u00e3 \u0111\u01b0\u1ee3c FDA ch\u1ea5p thu\u1eadn IND v\u00e0 ch\u1ec9 \u0111\u1ecbnh theo d\u00f5i nhanh. Nh\u00f3m \u0111\u1ea7u ti\u00ean \u0111\u00e3 b\u01b0\u1edbc v\u00e0o th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng giai \u0111o\u1ea1n I\/IIa v\u1edbi nh\u1eefng k\u1ebft qu\u1ea3 ban \u0111\u1ea7u \u0111\u1ea7y tri\u1ec3n v\u1ecdng. H\u00ecnh \u1ea3nh ch\u1ee5p PET sau 6, 12 v\u00e0 18 th\u00e1ng s\u1ebd gi\u00fap x\u00e1c \u0111\u1ecbnh dopamine trong n\u00e3o b\u1ec7nh nh\u00e2n c\u00f3 ph\u1ee5c h\u1ed3i hay kh\u00f4ng. B\u00ean c\u1ea1nh \u0111\u00f3, m\u1ed7i l\u00f4 t\u1ebf b\u00e0o \u0111\u01b0\u1ee3c m\u00f4 t\u1ea3 b\u1eb1ng thu\u1eadt to\u00e1n h\u1ecdc m\u00e1y \u0111\u01b0\u1ee3c \u0111\u00e0o t\u1ea1o tr\u00ean d\u1eef li\u1ec7u b\u1ed9 gen v\u00e0 phi\u00ean m\u00e3 m\u1edf r\u1ed9ng \u0111\u1ec3 d\u1ef1 \u0111o\u00e1n hi\u1ec7u qu\u1ea3 l\u00e2m s\u00e0ng, v\u1edbi hy v\u1ecdng c\u00f3 th\u1ec3 s\u1eed d\u1ee5ng cho m\u1ecdi b\u1ec7nh nh\u00e2n.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Lieu_phap_gen_dieu_tri_Parkinson_%E2%80%93_Kich_thich_su_tai_sinh_cua_te_bao_than_kinh\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>Li\u1ec7u ph\u00e1p gen \u0111i\u1ec1u tr\u1ecb Parkinson &#8211; K\u00edch th\u00edch s\u1ef1 t\u00e1i sinh c\u1ee7a t\u1ebf b\u00e0o th\u1ea7n kinh<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>Li\u1ec7u ph\u00e1p gen AB-1005<\/strong> \u0111\u01b0a gen cho y\u1ebfu t\u1ed1 dinh d\u01b0\u1ee1ng th\u1ea7n kinh c\u00f3 ngu\u1ed3n g\u1ed1c t\u1eeb t\u1ebf b\u00e0o th\u1ea7n kinh \u0111\u1ec7m (GDNF) tr\u1ef1c ti\u1ebfp v\u00e0o nh\u00e2n b\u00e8o-v\u00f9ng n\u00e3o b\u1ecb t\u1ed5n th\u01b0\u01a1ng n\u1eb7ng. \u0110i\u1ec1u n\u00e0y gi\u00fap b\u1ea3o v\u1ec7 v\u00e0 k\u00edch th\u00edch s\u1ef1 ph\u00e1t tri\u1ec3n m\u1edbi c\u1ee7a c\u00e1c \u0111\u1ea7u cu\u1ed1i th\u1ea7n kinh.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">K\u1ebft qu\u1ea3 th\u1eed nghi\u1ec7m giai \u0111o\u1ea1n I cho th\u1ea5y, li\u1ec7u ph\u00e1p gen AB-1005 kh\u00f4ng ch\u1ec9 an to\u00e0n m\u00e0 c\u00f2n gi\u00fap b\u1ec7nh nh\u00e2n gi\u1ea3m li\u1ec1u d\u00f9ng levodopa h\u00e0ng ng\u00e0y. \u0110\u1ed3ng th\u1eddi c\u00e1c tri\u1ec7u ch\u1ee9ng v\u1eadn \u0111\u1ed9ng v\u1eabn \u1ed5n \u0111\u1ecbnh trong nh\u00f3m b\u1ec7nh nh\u1eb9, c\u1ea3i thi\u1ec7n \u0111\u00e1ng k\u1ec3 trong nh\u00f3m b\u1ec7nh trung b\u00ecnh. FDA \u0111\u00e3 c\u1ea5p Quy ch\u1ebf theo d\u00f5i nhanh v\u00e0 hi\u1ec7n \u0111ang ti\u1ebfn h\u00e0nh nghi\u00ean c\u1ee9u m\u00f9 \u0111\u00f4i Giai \u0111o\u1ea1n II, nh\u1eb1m \u0111\u1ea9y nhanh ti\u1ebfn \u0111\u1ed9 ph\u00e1t tri\u1ec3n li\u1ec7u ph\u00e1p n\u00e0y.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Giam_su_hoat_dong_qua_muc_cua_cac_te_bao_than_kinh_van_dong\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>Gi\u1ea3m s\u1ef1 ho\u1ea1t \u0111\u1ed9ng qu\u00e1 m\u1ee9c c\u1ee7a c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh v\u1eadn \u0111\u1ed9ng<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">\u1ede b\u1ec7nh Parkinson, v\u00f9ng nh\u00e2n d\u01b0\u1edbi \u0111\u1ed3i (STN) b\u1ecb k\u00edch th\u00edch qu\u00e1 m\u1ee9c do thi\u1ebfu dopamine, d\u1eabn \u0111\u1ebfn c\u00e1c tri\u1ec7u ch\u1ee9ng v\u1eadn \u0111\u1ed9ng \u0111\u1eb7c tr\u01b0ng. MeiraGTx \u0111\u00e3 ph\u00e1t tri\u1ec3n&nbsp;<strong>li\u1ec7u ph\u00e1p gen AAV-GAD<\/strong>, s\u1eed d\u1ee5ng v\u1ecf b\u1ecdc virus li\u00ean k\u1ebft adeno (AAV) \u0111\u1ec3 \u0111\u01b0a gen (GAD) v\u00e0o STN. Ch\u00fang s\u1ebd s\u1ea3n xu\u1ea5t ch\u1ea5t \u1ee9c ch\u1ebf d\u1eabn truy\u1ec1n th\u1ea7n kinh (GABA). Li\u1ec7u ph\u00e1p gen AAV-GAD \u0111\u01b0\u1ee3c truy\u1ec1n v\u00e0o n\u00e3o b\u1eb1ng c\u00f9ng m\u1ed9t ph\u1eabu thu\u1eadt hi\u1ec7n \u0111ang \u0111\u01b0\u1ee3c s\u1eed d\u1ee5ng \u0111\u1ec3 k\u00edch th\u00edch n\u00e3o s\u00e2u, gi\u00fap l\u00e0m d\u1ecbu STN b\u1eb1ng \u0111i\u1ec7n.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">K\u1ebft qu\u1ea3 t\u1eeb c\u00e1c th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng giai \u0111o\u1ea1n I v\u00e0 II cho th\u1ea5y li\u1ec7u ph\u00e1p gen AAV-GAD l\u00e0 an to\u00e0n. Li\u1ec7u ph\u00e1p n\u00e0y c\u0169ng mang l\u1ea1i c\u1ea3i thi\u1ec7n r\u00f5 r\u1ec7t v\u1ec1 tri\u1ec7u ch\u1ee9ng v\u1eadn \u0111\u1ed9ng c\u0169ng nh\u01b0 ch\u1ea5t l\u01b0\u1ee3ng s\u1ed1ng. MeiraGTx \u0111\u00e3 th\u1ef1c hi\u1ec7n nghi\u00ean c\u1ee9u b\u1eafc c\u1ea7u theo h\u01b0\u1edbng d\u1eabn c\u1ee7a FDA. M\u1ee5c ti\u00eau l\u00e0 s\u1eed d\u1ee5ng v\u1eadt li\u1ec7u c\u1ee7a m\u00ecnh trong nghi\u00ean c\u1ee9u Giai \u0111o\u1ea1n III, t\u1eeb \u0111\u00f3 h\u1ed7 tr\u1ee3 vi\u1ec7c n\u1ed9p \u0111\u01a1n xin c\u1ea5p ph\u00e9p to\u00e0n c\u1ea7u v\u00e0 ti\u1ebfn g\u1ea7n h\u01a1n \u0111\u1ebfn m\u1ee5c ti\u00eau th\u01b0\u01a1ng m\u1ea1i h\u00f3a.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Lieu_phap_gen_dieu_tri_parkinson_khong_can_phau_thuat\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>Li\u1ec7u ph\u00e1p gen \u0111i\u1ec1u tr\u1ecb parkinson kh\u00f4ng c\u1ea7n ph\u1eabu thu\u1eadt<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">M\u1ed9t th\u00e1ch th\u1ee9c l\u1edbn trong li\u1ec7u ph\u00e1p gen l\u00e0 vi\u1ec7c \u0111\u01b0a c\u00e1c ph\u00e2n t\u1eed l\u1edbn v\u00e0o n\u00e3o qua h\u00e0ng r\u00e0o m\u00e1u n\u00e3o.<\/span><a target=\"_blank\" rel=\"noopener noreferrer\" href=\"https:\/\/www.voyagertherapeutics.com\/\"><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\"> Voyager Therapeutics&nbsp;<\/span><\/a><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">\u0111\u00e3 kh\u1eafc ph\u1ee5c b\u1eb1ng c\u00e1ch ph\u00e1t tri\u1ec3n gen GBA1 \u0111\u01b0\u1ee3c mang b\u1edfi m\u1ed9t v\u1ecf b\u1ecdc AAV c\u00f3 kh\u1ea3 n\u0103ng v\u01b0\u1ee3t qua h\u00e0ng r\u00e0o n\u00e0y. T\u1eeb \u0111\u00f3 cho ph\u00e9p \u0111\u01b0a gen v\u00e0o n\u00e3o m\u00e0 kh\u00f4ng c\u1ea7n ph\u1eabu thu\u1eadt.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>C\u00f4ng ngh\u1ec7 TRACER<\/strong> (chuy\u1ec3n h\u01b0\u1edbng h\u01b0\u1edbng \u0111\u1ed9ng c\u1ee7a AAV b\u1eb1ng c\u00e1ch bi\u1ec3u hi\u1ec7n RNA \u0111\u1eb7c hi\u1ec7u theo lo\u1ea1i t\u1ebf b\u00e0o) t\u1eadn d\u1ee5ng c\u00e1c c\u01a1 ch\u1ebf t\u1ef1 nhi\u00ean trong n\u00e3o \u0111\u1ec3 v\u1eadn chuy\u1ec3n c\u00e1c li\u1ec7u ph\u00e1p gen. H\u1ec7 th\u1ed1ng n\u00e0y kh\u00f4ng ch\u1ec9 gi\u00fap ph\u00e2n ph\u1ed1i li\u1ec7u ph\u00e1p gen di\u1ec7n r\u1ed9ng trong n\u00e3o m\u00e0 c\u00f2n gi\u1ea3m thi\u1ec3u \u1ea3nh h\u01b0\u1edfng \u0111\u1ebfn gan, m\u1edf ra tri\u1ec3n v\u1ecdng cho c\u00e1c \u1ee9ng d\u1ee5ng \u0111i\u1ec1u tr\u1ecb kh\u00e1c trong t\u01b0\u01a1ng lai.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Giam_tich_tu_Protein_%CE%B1-Synuclein\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>Gi\u1ea3m t\u00edch t\u1ee5 Protein \u03b1-Synuclein<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">M\u1ed9t d\u1ea5u hi\u1ec7u \u0111\u1eb7c tr\u01b0ng c\u1ee7a Parkinson l\u00e0 s\u1ef1 t\u00edch t\u1ee5 c\u1ee7a protein \u03b1-synuclein trong n\u00e3o.&nbsp;<\/span><a target=\"_blank\" rel=\"noopener noreferrer\" href=\"https:\/\/www.denalitherapeutics.com\/home\"><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\">Denali Therapeutics<\/span><\/a><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"> \u0111\u00e3 ph\u00e1t tri\u1ec3n ph\u01b0\u01a1ng ph\u00e1p v\u1eadn chuy\u1ec3n&nbsp;<strong>antisense oligonucleotide&nbsp;<\/strong>v\u01b0\u1ee3t qua h\u00e0ng r\u00e0o m\u00e1u n\u00e3o \u0111\u1ec3 gi\u1ea3m bi\u1ec3u hi\u1ec7n c\u1ee7a protein n\u00e0y.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Ph\u01b0\u01a1ng ti\u1ec7n v\u1eadn chuy\u1ec3n (TV) c\u1ee7a Denali \u0111\u01b0a antisense oligo v\u00e0o n\u00e3o b\u1eb1ng c\u00e1ch li\u00ean k\u1ebft v\u1edbi th\u1ee5 th\u1ec3 transferrin, nh\u1eb1m v\u1eadn chuy\u1ec3n protein transferrin mang s\u1eaft v\u00e0o n\u00e3o. K\u1ebft qu\u1ea3 th\u1eed nghi\u1ec7m l\u00e2m s\u00e0ng ban \u0111\u1ea7u cho th\u1ea5y, TV \u0111\u00e3 ch\u1ee9ng minh kh\u1ea3 n\u0103ng \u0111\u01b0a thu\u1ed1c v\u00e0o n\u00e3o m\u1ed9t c\u00e1ch hi\u1ec7u qu\u1ea3 v\u00e0 an to\u00e0n. T\u1eeb \u0111\u00f3, m\u1edf ra tri\u1ec3n v\u1ecdng m\u1edbi cho vi\u1ec7c \u0111i\u1ec1u tr\u1ecb c\u00e1c b\u1ec7nh th\u1ea7n kinh nh\u01b0 Parkinson v\u00e0 c\u00e1c lo\u1ea1i li\u1ec7u ph\u00e1p kh\u00e1c nhau.<\/span><\/p>\n<h2><span class=\"ez-toc-section\" id=\"Tuong_lai_dieu_tri_Parkinson\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>T\u01b0\u01a1ng lai \u0111i\u1ec1u tr\u1ecb Parkinson<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Nh\u1eefng ti\u1ebfn b\u1ed9 trong li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c v\u00e0 gen mang l\u1ea1i hy v\u1ecdng l\u1edbn lao cho h\u00e0ng tri\u1ec7u b\u1ec7nh nh\u00e2n Parkinson tr\u00ean to\u00e0n c\u1ea7u. T\u1eeb vi\u1ec7c thay th\u1ebf t\u1ebf b\u00e0o th\u1ea7n kinh m\u1ea5t m\u00e1t, b\u1ea3o v\u1ec7 t\u1ebf b\u00e0o hi\u1ec7n c\u00f3, cho \u0111\u1ebfn gi\u1ea3m tri\u1ec7u ch\u1ee9ng v\u00e0 ng\u0103n ng\u1eeba t\u1ed5n th\u01b0\u01a1ng th\u00eam.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">D\u00f9 v\u1eabn c\u00f2n m\u1ed9t ch\u1eb7ng \u0111\u01b0\u1eddng d\u00e0i \u0111\u1ec3 c\u00e1c li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c v\u00e0 li\u1ec7u ph\u00e1p gen \u0111i\u1ec1u tr\u1ecb Parkinson tr\u1edf th\u00e0nh ti\u00eau chu\u1ea9n, nh\u01b0ng ti\u1ec1m n\u0103ng c\u1ee7a ch\u00fang l\u00e0 kh\u00f4ng th\u1ec3 ph\u1ee7 nh\u1eadn. C\u00e1c li\u1ec7u ph\u00e1p ti\u00ean ti\u1ebfn n\u00e0y kh\u00f4ng ch\u1ec9 c\u1ea3i thi\u1ec7n ch\u1ea5t l\u01b0\u1ee3ng s\u1ed1ng m\u00e0 c\u00f2n h\u01b0\u1edbng \u0111\u1ebfn m\u1ee5c ti\u00eau l\u1edbn h\u01a1n l\u00e0 ch\u1eefa kh\u1ecfi Parkinson.<\/span><\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>Biogroup Vietnam&nbsp;<\/strong>chuy\u00ean cung c\u1ea5p thi\u1ebft b\u1ecb c\u00f9ng d\u1ecbch v\u1ee5 k\u1ef9 thu\u1eadt, v\u1eadt t\u01b0 v\u00e0 h\u00f3a ch\u1ea5t trong c\u00f4ng ngh\u1ec7 sinh h\u1ecdc, y sinh. Ch\u00fang t\u00f4i lu\u00f4n s\u1eb5n s\u00e0ng c\u00f9ng Qu\u00fd v\u1ecb \u1ee9ng d\u1ee5ng nh\u1eefng gi\u1ea3i ph\u00e1p \u0111i\u1ec1u tr\u1ecb m\u1edbi nh\u01b0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson. \u0110\u1ec3 \u0111\u01b0\u1ee3c t\u01b0 v\u1ea5n th\u00eam th\u00f4ng tin, Qu\u00fd v\u1ecb vui l\u00f2ng li\u00ean h\u1ec7 th\u00f4ng tin b\u00ean d\u01b0\u1edbi:<\/span><\/p>\n<p>&nbsp;<\/p>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">\ud83d\udce9&nbsp;<\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:13.999999999999998pt\"><strong>Th\u00f4ng tin li\u00ean h\u1ec7:<\/strong><\/span><\/p>\n<ul>\n<li><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>Website:<\/strong><\/span><a target=\"_blank\" rel=\"noopener noreferrer\" href=\"https:\/\/biogroupvietnam.com\/public\/lien-he\"><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">&nbsp;<\/span><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\">https:\/\/biogroupvietnam.com\/public\/lien-he<\/span><\/a><\/li>\n<li><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>Hotline:<\/strong> +84 963 621 421&nbsp;<\/span><\/li>\n<li><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\"><strong>Email:<\/strong>&nbsp;<\/span><a href=\"mailto:info@biogroupvietnam.vn\"><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\">info@biogroupvietnam.vn<\/span><\/a><\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<h2><span class=\"ez-toc-section\" id=\"Tai_lieu_tham_khao\"><\/span><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:20pt\"><strong>T\u00e0i li\u1ec7u tham kh\u1ea3o<\/strong><\/span><span class=\"ez-toc-section-end\"><\/span><\/h2>\n<p><span style=\"background-color:transparent;color:#000000;font-family:Arial,sans-serif;font-size:12pt\">Mansell, J. (2024, July 10). Gene and cell therapies take aim at Parkinson\u2019s disease. Genetic Engineering &amp; Biotechnology News.&nbsp;<\/span><a target=\"_blank\" rel=\"noopener noreferrer\" href=\"https:\/\/www.genengnews.com\/topics\/drug-discovery\/gene-and-cell-therapies-take-aim-at-parkinsons-disease\/\"><span style=\"background-color:transparent;color:#1155cc;font-family:Arial,sans-serif;font-size:12pt\">https:\/\/www.genengnews.com\/topics\/drug-discovery\/gene-and-cell-therapies-take-aim-at-parkinsons-disease\/<\/span><\/a><span style=\"background-color:transparent;color:#404654;font-family:Arial,sans-serif;font-size:12pt\">&nbsp;<\/span><\/p>\n","protected":false},"excerpt":{"rendered":"<p>Li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u tr\u1ecb Parkinson l\u00e0 c\u00e1c ph\u01b0\u01a1ng ph\u00e1p m\u1edbi \u00edt x\u00e2m l\u1ea5n nh\u01b0ng l\u00e2u d\u00e0i h\u1ee9a h\u1eb9n s\u1ebd ng\u0103n ch\u1eb7n v\u00e0 th\u1eadm ch\u00ed \u0111\u1ea3o ng\u01b0\u1ee3c qu\u00e1 tr\u00ecnh tho\u00e1i h\u00f3a th\u1ea7n kinh. &nbsp; Ph\u1ee5c h\u1ed3i ho\u1eb7c t\u00e1i t\u1ea1o t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ecb t\u1ed5n th\u01b0\u01a1ng l\u00e0 \u01b0u \u0111i\u1ec3m<\/p>\n<div class=\"klb-readmore post-buttons\"><a class=\"button rounded primary\" href=\"https:\/\/biogroupvietnam.com\/vi\/blog\/lieu-phap-gen-va-te-bao-huong-den-benh-parkinson\/\">Read More <i class=\"klbth-icon-right-arrow\"><\/i><\/a><\/div>\n","protected":false},"author":1,"featured_media":5971,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-4268","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-blog"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v26.8 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Li\u1ec7u ph\u00e1p gen v\u00e0 li\u1ec7u ph\u00e1p t\u1ebf b\u00e0o g\u1ed1c \u0111i\u1ec1u 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