\nStudiul publicat \u00een New England Journal of Medicine marcheaz\u0103 prima utilizare uman\u0103 a celulelor CAR-T editate genetic prin tehnica de editare a bazelor ADN, oferind speran\u0163\u0103 real\u0103 pacien\u0163ilor.<\/p>\n
\nTerapia se bazeaz\u0103 pe tehnica de editare a bazelor, o form\u0103 avansat\u0103 de CRISPR-Cas9, care permite modificarea precis\u0103 a literelor ADN f\u0103r\u0103 a t\u0103ia lan\u0163ul genetic. Echipa de cercet\u0103tori a efectuat patru modific\u0103ri asupra celulelor T donatoare:<\/p>\n
\nEliminarea receptorului TCR pentru a preveni respingerea celulelor de c\u0103tre organism.<\/p>\n
\n\u00cenl\u0103turarea HLA (antigen uman leucocitar), transform\u00e2nd celulele \u00een \u201cuniversale\u201d \u015fi compatibile pentru orice pacient.<\/p>\n
\n\u015etergerea proteinelor CD7 \u015fi CD52 de pe suprafa\u0163a celulelor T donatoare, astfel \u00eenc\u00e2t s\u0103 fie protejate \u00eempotriva medicamentului alemtuzumab \u015fi s\u0103 \u0163inteasc\u0103 exclusiv celulele canceroase.<\/p>\n
\nIntroducerea unui receptor CAR anti-CD7 care identific\u0103 \u015fi distruge celulele T-ALL.<\/p>\n
\nFaza ini\u0163ial\u0103 a studiului, desf\u0103\u015furat\u0103 \u00een 2022 pe 11 pacien\u0163i, majoritatea copii, a \u00eenregistrat o rat\u0103 de remisie ini\u0163ial\u0103 de 100%. Dintre ace\u015ftia, 82% au ajuns la remisie profund\u0103 sau nedetectabil\u0103 \u00een patru s\u0103pt\u0103m\u00e2ni, fiind eligibili pentru transplant de celule stem. Dup\u0103 tratament, aproximativ 64% dintre pacien\u0163i r\u0103m\u00e2n complet f\u0103r\u0103 boal\u0103, unii fiind vindeca\u0163i de mai bine de trei ani.<\/p>\n
\nPrimul pacient tratat, Alyssa, diagnosticat\u0103 la 13 ani cu T-ALL \u015fi f\u0103r\u0103 alte op\u0163iuni, este \u201ecancer-free\u201d din 2022 \u015fi tr\u0103ie\u015fte o via\u0163\u0103 normal\u0103 la 16 ani.<\/p>\n
Aceast\u0103 terapie nu doar ofer\u0103 o \u015fans\u0103 real\u0103 pacien\u0163ilor cu leucemie T-ALL considerat\u0103 incurabil\u0103, ci demonstreaz\u0103 \u015fi poten\u0163ialul celulelor universale s\u0103 accelereze \u015fi s\u0103 reduc\u0103 costurile tratamentului CAR-T \u00een viitor.<\/p>\n
\nPrintre efectele secundare se num\u0103r\u0103 reac\u0163iile imunitare exagerate, erup\u0163iile cutanate \u015fi sc\u0103derea temporar\u0103 a num\u0103rului de celule sanguine, dar beneficiile pe termen lung sunt promi\u0163\u0103toare.<\/p>\n
\n <\/p>\n","protected":false},"excerpt":{"rendered":"
O echip\u0103 de cercet\u0103tori de la University College London (UCL) \u015fi Great Ormond Street Hospital (GOSH) a anun\u0163at un progres revolu\u0163ionar \u00een tratarea leucemiei acute limfoblastice T (T-ALL), o form\u0103 de cancer al s\u00e2ngelui considerat\u0103 anterior incurabil\u0103.<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[510],"tags":[37958,42],"class_list":["post-243382","post","type-post","status-publish","format-standard","hentry","category-actualitate","tag-leucemie","tag-medicina"],"_links":{"self":[{"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=\/wp\/v2\/posts\/243382","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=243382"}],"version-history":[{"count":0,"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=\/wp\/v2\/posts\/243382\/revisions"}],"wp:attachment":[{"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=243382"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=243382"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/bm.dev.synology.me\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=243382"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}