{"id":6298,"date":"2019-04-02T09:30:01","date_gmt":"2019-04-02T02:30:01","guid":{"rendered":"https:\/\/www.vnras.com\/drug\/?p=6298"},"modified":"2019-04-06T07:38:53","modified_gmt":"2019-04-06T00:38:53","slug":"lieu-phap-gen-moi-trongdieu-tri-benh-mau-di-truyen-hiem-gap","status":"publish","type":"post","link":"https:\/\/vnras.com\/drug\/lieu-phap-gen-moi-trongdieu-tri-benh-mau-di-truyen-hiem-gap\/","title":{"rendered":"Li\u1ec7u ph\u00e1p gen m\u1edbi trong \u0111i\u1ec1u tr\u1ecb b\u1ec7nh m\u00e1u di truy\u1ec1n hi\u1ebfm g\u1eb7p"},"content":{"rendered":"<h2>EMA \u0111\u00e3 \u0111\u1ec1 ngh\u1ecb c\u1ea5p gi\u1ea5y ph\u00e9p l\u01b0u h\u00e0nh t\u1ea1i Li\u00ean minh Ch\u00e2u \u00c2u cho m\u1ed9t s\u1ea3n ph\u1ea9m bi\u1ebfn \u0111\u1ed5i gen s\u1eed d\u1ee5ng \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh beta-thalassemia, m\u1ed9t b\u1ec7nh m\u00e1u di truy\u1ec1n hi\u1ebfm g\u1eb7p g\u00e2y thi\u1ebfu m\u00e1u nghi\u00eam tr\u1ecdng.<\/h2>\n<p>Zynteglo s\u1eed d\u1ee5ng \u1edf ng\u01b0\u1eddi l\u1edbn v\u00e0 thanh thi\u1ebfu ni\u00ean t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean, nh\u1eefng ng\u01b0\u1eddi c\u1ea7n truy\u1ec1n m\u00e1u th\u01b0\u1eddng xuy\u00ean \u0111\u1ec3 ki\u1ec3m so\u00e1t b\u1ec7nh v\u00e0 kh\u00f4ng c\u00f3 ng\u01b0\u1eddi hi\u1ebfn t\u1eb7ng ph\u00f9 h\u1ee3p \u0111\u1ec3 gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c.<\/p>\n<p>B\u1ec7nh nh\u00e2n m\u1eafc <strong>b\u1ec7nh beta-thalassemia<\/strong> kh\u00f4ng th\u1ec3 s\u1ea3n xu\u1ea5t \u0111\u1ee7 beta-globin, th\u00e0nh ph\u1ea7n ch\u00ednh c\u1ee7a hemoglobin, protein mang oxy trong m\u00e1u t\u1eeb ph\u1ed5i \u0111\u1ebfn c\u00e1c c\u01a1 quanc\u00f2n l\u1ea1i trong c\u01a1 th\u1ec3. H\u1eadu qu\u1ea3 l\u00e0 b\u1ec7nh nh\u00e2n c\u00f3 s\u1ed1 l\u01b0\u1ee3ng h\u1ed3ng c\u1ea7u \u00edt h\u01a1n nhi\u1ec1u so v\u1edbi b\u00ecnh th\u01b0\u1eddng v\u00e0 d\u1eabn \u0111\u1ebfn thi\u1ebfu m\u00e1u n\u1eb7ng m\u00e3n t\u00ednh.<\/p>\n<p>Nh\u1eefng b\u1ec7nh nh\u00e2n n\u00e0y th\u01b0\u1eddng ph\u1ea3i truy\u1ec1n m\u00e1u su\u1ed1t \u0111\u1eddi v\u00ec thi\u1ebfu m\u00e1u. Truy\u1ec1n m\u00e1u k\u00e9o d\u00e0i c\u00f3 th\u1ec3 g\u00e2y qu\u00e1 t\u1ea3i s\u1eaft, do \u0111\u00f3 b\u1ec7nh nh\u00e2n c\u1ea7n d\u00f9ng thu\u1ed1c \u0111\u1ec3 lo\u1ea1i b\u1ecf l\u01b0\u1ee3ng s\u1eaft d\u01b0 th\u1eeba. Cho \u0111\u1ebfn nay, l\u1ef1a ch\u1ecdn \u0111i\u1ec1u tr\u1ecb duy nh\u1ea5t l\u00e0 gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c t\u1eeb m\u1ed9t ng\u01b0\u1eddi hi\u1ebfn t\u1eb7ng kh\u1ecfe m\u1ea1nh. Tuy nhi\u00ean, trong tr\u01b0\u1eddng h\u1ee3p kh\u00f4ng c\u00f3 ng\u01b0\u1eddi hi\u1ebfn t\u1eb7ng ph\u00f9 h\u1ee3p trong gia \u0111\u00ecnh, vi\u1ec7c t\u00ecm ki\u1ebfm ng\u01b0\u1eddi hi\u1ebfn ph\u00f9 h\u1ee3p th\u01b0\u1eddng kh\u00f3 kh\u0103n v\u00e0 vi\u1ec7c gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c v\u1edbi ng\u01b0\u1eddi hi\u1ebfn kh\u00f4ng c\u00f9ng huy\u1ebft th\u1ed1ng c\u00f3 th\u1ec3 x\u1ea3y \u0111\u1ebfn c\u00e1c t\u00e1c d\u1ee5ng ph\u1ee5 v\u00e0 ph\u1ea3n \u1ee9ng nghi\u00eam tr\u1ecdng. Do \u0111\u00f3, nh\u1eefng b\u1ec7nh nh\u00e2n ph\u1ee5 thu\u1ed9c v\u00e0o truy\u1ec1n m\u00e1u \u0111\u1ec3 ki\u1ec3m so\u00e1t beta-thalassemia r\u1ea5t c\u1ea7n c\u00e1c ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb m\u1edbi.<\/p>\n<p>Zynteglo l\u00e0 m\u1ed9t l\u1ef1a ch\u1ecdn tr\u1ecb li\u1ec7u m\u1edbi cho nh\u1eefng b\u1ec7nh nh\u00e2n kh\u00f4ng c\u00f3 ng\u01b0\u1eddi hi\u1ebfn t\u1eb7ng ph\u00f9 h\u1ee3p \u0111\u1ec3 gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c. S\u1eed d\u1ee5ng m\u1ed9t vec t\u01a1 lentivirus, n\u00f3 b\u1ed5 sung c\u00e1c b\u1ea3n sao ch\u1ee9c n\u0103ng c\u1ee7a gen-globin \u0111\u00e3 \u0111\u01b0\u1ee3c s\u1eeda \u0111\u1ed5i v\u00e0o t\u1ebf b\u00e0o g\u1ed1c c\u1ee7a b\u1ec7nh nh\u00e2n, t\u1eeb \u0111\u00f3 gi\u1ea3i quy\u1ebft nguy\u00ean nh\u00e2n di truy\u1ec1n ti\u1ec1m \u1ea9n c\u1ee7a b\u1ec7nh.<\/p>\n<p>Zynteglo ch\u1ec9 \u0111\u01b0\u1ee3c qu\u1ea3n l\u00fd t\u1ea1i m\u1ed9t trung t\u00e2m \u0111i\u1ec1u tr\u1ecb \u0111\u1ee7 \u0111i\u1ec1u ki\u1ec7n b\u1edfi m\u1ed9t b\u00e1c s\u0129 c\u00f3 kinh nghi\u1ec7m v\u1ec1 c\u1ea5y gh\u00e9p t\u1ebf b\u00e0o g\u1ed1c v\u00e0 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh nh\u00e2n b\u1ecb beta-thalassemia.<\/p>\n<p>Trong hai nghi\u00ean c\u1ee9u ch\u00ednh ch\u1ee9ng minh t\u00e1c d\u1ee5ng c\u1ee7a Zynteglo, ng\u01b0\u1eddi ta \u0111\u00e3 ch\u1ec9 ra r\u1eb1ng ph\u1ea7n l\u1edbn b\u1ec7nh nh\u00e2n kh\u00f4ng c\u00f3 ki\u1ec3u gen \u03b20 \/ \u03b20 \u0111\u01b0\u1ee3c \u0111i\u1ec1u tr\u1ecb b\u1eb1ng Zynteglo kh\u00f4ng c\u1ea7n truy\u1ec1n m\u00e1u th\u01b0\u1eddng xuy\u00ean n\u1eefa.<\/p>\n<p>C\u00e1c t\u00e1c d\u1ee5ng ph\u1ee5 ph\u1ed5 bi\u1ebfn nh\u1ea5t l\u00e0 gi\u1ea3m ti\u1ec3u c\u1ea7u,\u0111au b\u1ee5ng, \u0111au ng\u1ef1c kh\u00f4ng do nguy\u00ean nh\u00e2n \u1edf tim, \u0111au \u1edf t\u1ee9 chi, kh\u00f3 th\u1edf v\u00e0 n\u00f3ng b\u1eebng.<\/p>\n<p>Do Zynteglo gi\u1ea3i quy\u1ebft nhu c\u1ea7u \u0111i\u1ec1u tr\u1ecb ch\u01b0a \u0111\u01b0\u1ee3c \u0111\u00e1p \u1ee9ng, n\u00ean thu\u1ed1c n\u00e0y \u0111\u01b0\u1ee3c c\u00e1c nh\u00e0 ph\u00e1t tri\u1ec3n thu\u1ed1c m\u1edbi c\u00f3 tri\u1ec3n v\u1ecdng t\u1eadp trung nghi\u00ean c\u1ee9u. \u0110i\u1ec1u n\u00e0y th\u00fac \u0111\u1ea9y m\u1ea1nh vi\u1ec7c ti\u1ebfn h\u00e0nh c\u00e1c nghi\u00ean c\u1ee9u ch\u1ee9ng minh c\u00e1c l\u1ee3i \u00edch v\u00e0 r\u1ee7i ro do thu\u1ed1c , cho ph\u00e9p \u0111\u00e1nh gi\u00e1 nhanh Zynteglo trong 150 ng\u00e0y, th\u1eddi \u0111i\u1ec3m \u0111\u00e1nh gi\u00e1 s\u1ea3n ph\u1ea9m thu\u1ed1c \u0111i\u1ec1u tr\u1ecb ti\u00ean ti\u1ebfn (ATMP) nhanh nh\u1ea5t cho \u0111\u1ebfn nay.<\/p>\n<p>B\u1edfi v\u00ec Zynteglo l\u00e0 m\u1ed9t ATMP, n\u00f3 \u0111\u01b0\u1ee3c \u0111\u00e1nh gi\u00e1 b\u1edfi \u1ee6y ban Tr\u1ecb li\u1ec7u N\u00e2ng cao (CAT), \u1ee7y ban chuy\u00ean gia c\u1ee7a C\u01a1 quan v\u1ec1 c\u00e1c lo\u1ea1i thu\u1ed1c d\u1ef1a tr\u00ean t\u1ebf b\u00e0o v\u00e0 gen.<\/p>\n<p>Tr\u00ean c\u01a1 s\u1edf \u0111\u00e1nh gi\u00e1 c\u1ee7a CAT v\u00e0 nhi\u1ec1u\u00fd ki\u1ebfn \u200b\u200bt\u00edch c\u1ef1c,CHMP\u0111\u00e3 \u0111\u1ec1 ngh\u1ecb ph\u00ea duy\u1ec7t c\u00f3 \u0111i\u1ec1u ki\u1ec7n cho lo\u1ea1i thu\u1ed1c n\u00e0y. \u0110\u00e2y l\u00e0 m\u1ed9t trong nh\u1eefng c\u01a1 ch\u1ebf qu\u1ea3n l\u00fd c\u1ee7a EU nh\u1eb1m t\u1ea1o \u0111i\u1ec1u ki\u1ec7n ti\u1ebfp c\u1eadn s\u1edbm v\u1edbi c\u00e1c lo\u1ea1i thu\u1ed1c gi\u1ea3i quy\u1ebft c\u00e1c nhu c\u1ea7u y t\u1ebf ch\u01b0a \u0111\u01b0\u1ee3c \u0111\u00e1p \u1ee9ng. Ki\u1ec3u ph\u00ea duy\u1ec7t n\u00e0y cho ph\u00e9pC\u01a1 quan n\u00e0y c\u1ea5p ph\u00e9p l\u01b0u h\u00e0nh v\u1edbi c\u00e1c lo\u1ea1i thu\u1ed1c c\u00f2n thi\u1ebfu d\u1eef li\u1ec7u ch\u1ee9ng minh an to\u00e0n, trong tr\u01b0\u1eddng h\u1ee3p l\u1ee3i \u00edch c\u1ee7a thu\u1ed1c mang l\u1ea1i ngay l\u1eadp t\u1ee9c \u0111\u1ed1i v\u1edbi b\u1ec7nh nh\u00e2n v\u01b0\u1ee3t tr\u1ed9i h\u01a1n c\u00e1c r\u1ee7i ro c\u00f3 th\u1ec3.<\/p>\n<p>Ngu\u1ed3n: <a href=\"https:\/\/www.ema.europa.eu\/en\/news\/new-gene-therapy-treat-rare-inherited-blood-condition\" rel=\"noopener\">https:\/\/www.ema.europa.eu\/en\/news\/new-gene-therapy-treat-rare-inherited-blood-condition<\/a> D\u1ecbch b\u1edfi Trung T\u00e2m C\u1ea3nh Gi\u00e1c D\u01b0\u1ee3c Qu\u1ed1c Gia.<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n<p>&nbsp;<\/p>\n","protected":false},"excerpt":{"rendered":"<p>EMA \u0111\u00e3 \u0111\u1ec1 ngh\u1ecb c\u1ea5p gi\u1ea5y ph\u00e9p l\u01b0u h\u00e0nh t\u1ea1i Li\u00ean minh Ch\u00e2u \u00c2u cho m\u1ed9t s\u1ea3n ph\u1ea9m bi\u1ebfn \u0111\u1ed5i gen s\u1eed d\u1ee5ng \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb b\u1ec7nh beta-thalassemia, m\u1ed9t b\u1ec7nh m\u00e1u di truy\u1ec1n hi\u1ebfm g\u1eb7p g\u00e2y thi\u1ebfu m\u00e1u nghi\u00eam tr\u1ecdng. Zynteglo s\u1eed d\u1ee5ng \u1edf ng\u01b0\u1eddi l\u1edbn v\u00e0 thanh thi\u1ebfu ni\u00ean t\u1eeb 12 tu\u1ed5i tr\u1edf l\u00ean, [&#8230;]\n","protected":false},"author":1,"featured_media":6299,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[1],"tags":[],"class_list":["post-6298","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-y-te-suc-khoe"],"acf":[],"_links":{"self":[{"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/posts\/6298","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/comments?post=6298"}],"version-history":[{"count":0,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/posts\/6298\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/media\/6299"}],"wp:attachment":[{"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/media?parent=6298"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/categories?post=6298"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/vnras.com\/drug\/wp-json\/wp\/v2\/tags?post=6298"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}