{"id":14268,"date":"2022-05-11T11:04:37","date_gmt":"2022-05-11T09:04:37","guid":{"rendered":"https:\/\/havas.cz\/?post_type=pressroom&p=14268"},"modified":"2022-05-11T11:04:38","modified_gmt":"2022-05-11T09:04:38","slug":"genova-a-bunecna-terapie-nastup-nove-generace-lecby","status":"publish","type":"pressroom","link":"https:\/\/havas.cz\/en\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/","title":{"rendered":"Genov\u00e1 a bun\u011b\u010dn\u00e1 terapie: n\u00e1stup nov\u00e9 generace l\u00e9\u010dby"},"content":{"rendered":"\n

\u201e<\/em>U genov\u00e9 a bun\u011b\u010dn\u00e9 terapie se nejedn\u00e1 o b\u011b\u017en\u00e9 tabletky, jak\u00e9 zn\u00e1me doposud. L\u00e9\u010dba je vyvinut\u00e1 na m\u00edru konkr\u00e9tn\u00edmu pacientovi, \u010dasto z jeho vlastn\u00edho biologick\u00e9ho materi\u00e1lu. Vyu\u017e\u00edv\u00e1 geny a bu\u0148ky pro p\u0159eprogramov\u00e1n\u00ed t\u011bla tak, aby bojovalo s nemoc\u00ed. Efekt l\u00e9\u010dby m\u016f\u017ee b\u00fdt trval\u00fd po pod\u00e1n\u00ed jedin\u00e9 d\u00e1vky,\u201c <\/em>vysv\u011btluje prof. MUDr. Milan Macek jr., DrSc., MHA, p\u0159ednosta \u00dastavu biologie a l\u00e9ka\u0159sk\u00e9 genetiky 2. LF a FN Motol. Vyu\u017eit\u00ed obou typ\u016f terapi\u00ed je \u0161irok\u00e9 a pomoci m\u016f\u017ee nap\u0159\u00edklad u onemocn\u011bn\u00ed krve a o\u010d\u00ed, kardiovaskul\u00e1rn\u00edch i neurologick\u00fdch onemocn\u011bn\u00ed a u n\u011bkter\u00fdch typ\u016f n\u00e1dorov\u00fdch onemocn\u011bn\u00ed.4<\/sup><\/p>\n\n\n\n

Co jsou to bu\u0148ky a geny<\/strong><\/p>\n\n\n\n

Bu\u0148ky jsou z\u00e1kladn\u00ed stavebn\u00ed jednotkou v\u0161ech \u017eiv\u00fdch organism\u016f. Hluboko v nich se nach\u00e1zej\u00ed geny, mal\u00e9 \u00faseky DNA, kter\u00e9 nesou genetickou informaci a n\u00e1vod pro t\u011blo, jak si vyr\u00e1b\u011bt b\u00edlkoviny. Jedn\u00e1 se o b\u00edlkoviny, kter\u00e9 maj\u00ed v t\u011ble nespo\u010det \u00fakol\u016f \u2013 od role stavebn\u00edch prvk\u016f po hlavn\u00ed regulaci t\u011blesn\u00fdch funkc\u00ed.5<\/sup> \u201e<\/em>Geny nesou z\u00e1kladn\u00ed informace, kter\u00e9 d\u011bd\u00edme od na\u0161ich rodi\u010d\u016f. V n\u011bkter\u00fdch situac\u00edch m\u016f\u017ee doch\u00e1zet k mutac\u00edm, kter\u00e9 zp\u016fsobuj\u00ed vz\u00e1cn\u00e1 onemocn\u011bn\u00ed. S t\u011bmi m\u016f\u017ee nov\u011b pom\u00e1hat genov\u00e1 a bun\u011b\u010dn\u00e1 terapie, kter\u00e1 genetick\u00e9 defekty c\u00edlen\u011b \u0159e\u0161\u00ed,\u201c<\/em> \u0159\u00edk\u00e1 profesor Macek.<\/p>\n\n\n\n

Jak funguje bun\u011b\u010dn\u00e1 terapie<\/strong><\/p>\n\n\n\n

C\u00edlem bun\u011b\u010dn\u00e9 terapie je obnovit nebo pozm\u011bnit ur\u010dit\u00fd soubor bun\u011bk v na\u0161em t\u011ble.1<\/sup> P\u0159i terapii jsou vyu\u017e\u00edv\u00e1ny bu\u0148ky l\u00e9\u010den\u00e9ho pacienta nebo d\u00e1rce. Terapie s vyu\u017eit\u00edm d\u00e1rcovsk\u00fdch bun\u011bk jsou ji\u017e zn\u00e1m\u00e9 nap\u0159\u00edklad v podob\u011b transplantace kostn\u00ed d\u0159en\u011b u leuk\u00e9mie. P\u0159i t\u00e9to procedu\u0159e ale nejsou bu\u0148ky od d\u00e1rce nijak upraven\u00e9. Nov\u00e9 terapie vyu\u017e\u00edvaj\u00ed vlastn\u00edch bun\u011bk pacienta nebo d\u00e1rce. Ty jsou po odebr\u00e1n\u00ed upraveny v laborato\u0159i tak, aby v t\u011ble splnily ur\u010ditou l\u00e9\u010debnou funkci, n\u00e1sledn\u011b jsou vr\u00e1ceny zp\u011bt do t\u011bla.3,6<\/sup><\/p>\n\n\n\n

Jak funguje genov\u00e1 terapie<\/strong><\/p>\n\n\n\n

Genov\u00e1 terapie l\u00e9\u010d\u00ed onemocn\u011bn\u00ed prost\u0159ednictv\u00edm gen\u016f, a to n\u011bkolika zp\u016fsoby: p\u0159id\u00e1n\u00edm nov\u00e9ho genu do bun\u011bk pacienta, nahrazen\u00edm naru\u0161en\u00fdch gen\u016f nebo zablokov\u00e1n\u00edm ne\u017e\u00e1douc\u00ed aktivity ur\u010dit\u00fdch gen\u016f. Aby se nov\u00e9 upraven\u00e9 geny dostaly do bun\u011bk pacienta, vyu\u017e\u00edvaj\u00ed se speci\u00e1ln\u00ed p\u0159ena\u0161e\u010de, tzv. vektory. Vektory jsou \u010dasto viry zbaven\u00e9 vlastn\u00ed aktivity \u2013 nemohou tak vyvolat \u017e\u00e1dn\u00e1 onemocn\u011bn\u00ed. Pou\u017eity jsou pouze jako schr\u00e1nka, kter\u00e1 doprav\u00ed nov\u00fd gen do bun\u011bk pacienta.  Nov\u00fd funguj\u00edc\u00ed gen uvnit\u0159 bun\u011bk zajist\u00ed, \u017ee t\u011blo dok\u00e1\u017ee spr\u00e1vn\u011b tvo\u0159it b\u00edlkoviny pot\u0159ebn\u00e9 pro vykon\u00e1v\u00e1n\u00ed specifick\u00fdch funkc\u00ed.3<\/sup><\/p>\n\n\n\n

Rozd\u00edly oproti b\u011b\u017en\u00fdm l\u00e9\u010debn\u00fdm metod\u00e1m<\/strong><\/p>\n\n\n\n

\u201eU b\u011b\u017en\u00fdch terapi\u00ed u\u017e\u00edv\u00e1me k l\u00e9\u010db\u011b tablety, injekce nebo infuze, kter\u00e9 mohou ztratit \u00fa\u010dinnost, jakmile je pacient p\u0159estane u\u017e\u00edvat. Pom\u00e1haj\u00ed dlouhodob\u011b zvl\u00e1dat projevy onemocn\u011bn\u00ed, n\u011bkter\u00e9 z\u00e1va\u017en\u00e9 choroby ale vyl\u00e9\u010dit neum\u011bj\u00ed. Bun\u011b\u010dn\u00e1 a genov\u00e1 terapie funguj\u00ed zcela odli\u0161n\u011b. Zasahuj\u00ed do samotn\u00e9 p\u0159\u00ed\u010diny vzniku onemocn\u011bn\u00ed \u2013 vadn\u00e9ho genu nebo bun\u011bk. Maj\u00ed tak potenci\u00e1l tato onemocn\u011bn\u00ed nejenom l\u00e9\u010dit, ale v n\u011bkter\u00fdch p\u0159\u00edpadech i vyl\u00e9\u010dit,\u201c<\/em> \u0159\u00edk\u00e1 profesor Macek. U velmi komplikovan\u00fdch onemocn\u011bn\u00ed je mo\u017enost\u00ed kombinovat ob\u011b terapie \u2013 bun\u011b\u010dnou i genovou.3<\/sup><\/p>\n\n\n\n

Vz\u00e1cn\u00e1 a genetick\u00e1 onemocn\u011bn\u00ed<\/strong><\/p>\n\n\n\n

Nov\u00e1 generace terapi\u00ed se zam\u011b\u0159uje na genetick\u00e1 onemocn\u011bn\u00ed i z\u00edskan\u00e9 choroby, \u010dasto velmi z\u00e1va\u017en\u00e9 a\u017e \u017eivot ohro\u017euj\u00edc\u00ed.3<\/sup> M\u016f\u017ee se zd\u00e1t, \u017ee tato onemocn\u011bn\u00ed nejsou nijak \u010dast\u00e1. Pravdou ale je, \u017ee nejsou tak vz\u00e1cn\u00e1, jak si podle n\u00e1zvu m\u016f\u017eeme myslet.7<\/sup> Podle r\u016fzn\u00fdch odhad\u016f trp\u00ed miliony lid\u00ed na cel\u00e9m sv\u011bt\u011b n\u011bkter\u00fdm ze zhruba 5 000\u20138 000 vz\u00e1cn\u00fdch onemocn\u011bn\u00ed, z nich\u017e zhruba 80 % je geneticky podm\u00edn\u011bno.8,9<\/sup><\/p>\n\n\n\n

Kontakt pro m\u00e9dia: <\/p>\n\n\n\n

Krist\u00fdna Hudeov\u00e1, PR Manager, Havas, 727 818 975, kristyna.hudeova@havaspr.com<\/a><\/p>\n\n\n\n

Zdroje: <\/strong><\/p>\n\n\n\n

  1. FRIEDMAN, T. A brief history of gene therapy. Nat Genet<\/em>. 1992; 2: 93\u201398.<\/li>
  2. Harvard Stem Cell Institute. Cell Therapies<\/em> [online]. [Cit. 26. 11. 2020]. Dostupn\u00e9 z: https:\/\/hsci.harvard.edu\/translation\/what-are-drugs-5-cell-therapies<\/a><\/li>
  3. American Society of Gene & Cell Therapy. Different approaches<\/em> [online]. 11. 5. 2021 [cit. 5. 10. 2021]. Dostupn\u00e9 z: https:\/\/www.asgct.org\/education\/different-approaches<\/a>.<\/li>
  4. O\u2019REILLY, M., KOHN, D. B., BARTLETT, J., et al. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 2012. Hum Gene Ther<\/em>. 2013; 24(4): 355\u2013362.<\/li>
  5. National Institutes of Health (NIH) U.S. National Library of Medicine. What is a gene?<\/em> [online]. [Cit. 5. 10. 2021]. Dostupn\u00e9 z: https:\/\/ghr.nlm.nih.gov\/primer\/basics\/gene. <\/li>
  6. Typy, d\u00e1rci a indikace k transplantac\u00edm. In: Linkos.cz<\/em> [online]. [Cit. 5. 10. 2021]. Dostupn\u00e9 z: https:\/\/www.linkos.cz\/pacient-a-rodina\/lecba\/jak-se-lecit\/transplantace-krvetvornych-bunek\/typy-darci-a-indikace-k-transplantacim\/<\/a><\/li>
  7. World Health Organization. Genes and human diseases<\/em> [online]. [Cit. 5. 10. 2021]. Dostupn\u00e9 z: http:\/\/www.who.int\/genomics\/public\/geneticdiseases\/en\/index2.html<\/a><\/li>
  8. HAENDEL, Melissa, VASILEVSKY, Nicole, DEEPAK, Unni, et al. How many rare diseases are there? Nature Reviews<\/em> Drug Discovery<\/em> [online]. 2020; 19: 77\u201378 [cit. 21. 10. 2021] Dostupn\u00e9 z https:\/\/www.nature.com\/articles\/d41573-019-00180-y?linkId=76707460<\/a><\/li>
  9. America\u2019s Biopharmaceutical Companies. Rare disease by the numbers<\/em> [online]. Dostupn\u00e9 z: https:\/\/innovation.org\/about-us\/commitment\/research-discovery\/rare-disease-numbers<\/a><\/li><\/ol>\n","protected":false},"excerpt":{"rendered":"

    Po v\u00edce ne\u017e 50 letech v\u00fdzkumu p\u0159in\u00e1\u0161ej\u00ed bun\u011b\u010dn\u00e9 a genov\u00e9 terapie novou kapitolu \u2013 nejen do medic\u00edny, ale tak\u00e9 do \u017eivot\u016f lid\u00ed se vz\u00e1cn\u00fdm onemocn\u011bn\u00edm a jejich rodin.1,2 Tyto terapie jsou vyv\u00edjeny s c\u00edlem p\u0159edch\u00e1zet, l\u00e9\u010dit nebo potenci\u00e1ln\u011b vyl\u00e9\u010dit z\u00e1va\u017en\u00e1 onemocn\u011bn\u00ed.3 Za\u010d\u00ednaj\u00ed se vyu\u017e\u00edvat jak ve sv\u011bt\u011b, tak i v \u010cesk\u00e9 republice. Dok\u00e1\u017eou pomoci mnohdy jen jednou aplikac\u00ed l\u00e9ku, kter\u00e1 p\u016fsob\u00ed dlouhodob\u011b, n\u011bkdy na cel\u00fd \u017eivot. Nov\u00e1 generace terapi\u00ed tak p\u0159in\u00e1\u0161\u00ed mo\u017en\u00e9 \u0159e\u0161en\u00ed pro pacienty s doposud obt\u00ed\u017en\u011b l\u00e9\u010diteln\u00fdmi nebo nevyl\u00e9\u010diteln\u00fdmi onemocn\u011bn\u00edmi.1<\/p>\n","protected":false},"author":48,"featured_media":14324,"menu_order":0,"template":"","meta":{"_links_to":"","_links_to_target":""},"pressroom_category":[],"pressroom_tag":[204],"class_list":["post-14268","pressroom","type-pressroom","status-publish","has-post-thumbnail","hentry","pressroom_tag-zdravi"],"yoast_head":"\nGenov\u00e1 a bun\u011b\u010dn\u00e1 terapie: n\u00e1stup nov\u00e9 generace l\u00e9\u010dby - Havas Prague<\/title>\n<meta name=\"description\" content=\"Po v\u00edce ne\u017e 50 letech v\u00fdzkumu p\u0159in\u00e1\u0161ej\u00ed bun\u011b\u010dn\u00e9 a genov\u00e9 terapie novou kapitolu \u2013 nejen do medic\u00edny, ale tak\u00e9 do \u017eivot\u016f lid\u00ed se vz\u00e1cn\u00fdm onemocn\u011bn\u00edm a jejich rodin.1,2 Tyto terapie jsou vyv\u00edjeny s c\u00edlem p\u0159edch\u00e1zet, l\u00e9\u010dit nebo potenci\u00e1ln\u011b vyl\u00e9\u010dit z\u00e1va\u017en\u00e1 onemocn\u011bn\u00ed.3 Za\u010d\u00ednaj\u00ed se vyu\u017e\u00edvat jak ve sv\u011bt\u011b, tak i v \u010cesk\u00e9 republice. Dok\u00e1\u017eou pomoci mnohdy jen jednou aplikac\u00ed l\u00e9ku, kter\u00e1 p\u016fsob\u00ed dlouhodob\u011b, n\u011bkdy na cel\u00fd \u017eivot. Nov\u00e1 generace terapi\u00ed tak p\u0159in\u00e1\u0161\u00ed mo\u017en\u00e9 \u0159e\u0161en\u00ed pro pacienty s doposud obt\u00ed\u017en\u011b l\u00e9\u010diteln\u00fdmi nebo nevyl\u00e9\u010diteln\u00fdmi onemocn\u011bn\u00edmi.1\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Genov\u00e1 a bun\u011b\u010dn\u00e1 terapie: n\u00e1stup nov\u00e9 generace l\u00e9\u010dby - Havas Prague\" \/>\n<meta property=\"og:description\" content=\"Po v\u00edce ne\u017e 50 letech v\u00fdzkumu p\u0159in\u00e1\u0161ej\u00ed bun\u011b\u010dn\u00e9 a genov\u00e9 terapie novou kapitolu \u2013 nejen do medic\u00edny, ale tak\u00e9 do \u017eivot\u016f lid\u00ed se vz\u00e1cn\u00fdm onemocn\u011bn\u00edm a jejich rodin.1,2 Tyto terapie jsou vyv\u00edjeny s c\u00edlem p\u0159edch\u00e1zet, l\u00e9\u010dit nebo potenci\u00e1ln\u011b vyl\u00e9\u010dit z\u00e1va\u017en\u00e1 onemocn\u011bn\u00ed.3 Za\u010d\u00ednaj\u00ed se vyu\u017e\u00edvat jak ve sv\u011bt\u011b, tak i v \u010cesk\u00e9 republice. 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Nov\u00e1 generace terapi\u00ed tak p\u0159in\u00e1\u0161\u00ed mo\u017en\u00e9 \u0159e\u0161en\u00ed pro pacienty s doposud obt\u00ed\u017en\u011b l\u00e9\u010diteln\u00fdmi nebo nevyl\u00e9\u010diteln\u00fdmi onemocn\u011bn\u00edmi.1\" \/>\n<meta property=\"og:url\" content=\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/\" \/>\n<meta property=\"og:site_name\" content=\"Havas Prague\" \/>\n<meta property=\"article:modified_time\" content=\"2022-05-11T09:04:38+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/havas.cz\/wp-content\/uploads\/2022\/05\/600x305-tod6897.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"600\" \/>\n\t<meta property=\"og:image:height\" content=\"305\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data1\" content=\"4 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/\",\"url\":\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/\",\"name\":\"Genov\u00e1 a bun\u011b\u010dn\u00e1 terapie: n\u00e1stup nov\u00e9 generace l\u00e9\u010dby - Havas Prague\",\"isPartOf\":{\"@id\":\"https:\/\/havas.cz\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/#primaryimage\"},\"image\":{\"@id\":\"https:\/\/havas.cz\/pressroom\/genova-a-bunecna-terapie-nastup-nove-generace-lecby\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/havas.cz\/wp-content\/uploads\/2022\/05\/600x305-tod6897.jpg\",\"datePublished\":\"2022-05-11T09:04:37+00:00\",\"dateModified\":\"2022-05-11T09:04:38+00:00\",\"description\":\"Po v\u00edce ne\u017e 50 letech v\u00fdzkumu p\u0159in\u00e1\u0161ej\u00ed bun\u011b\u010dn\u00e9 a genov\u00e9 terapie novou kapitolu \u2013 nejen do medic\u00edny, ale tak\u00e9 do \u017eivot\u016f lid\u00ed se vz\u00e1cn\u00fdm onemocn\u011bn\u00edm a jejich rodin.1,2 Tyto terapie jsou vyv\u00edjeny s c\u00edlem p\u0159edch\u00e1zet, l\u00e9\u010dit nebo potenci\u00e1ln\u011b vyl\u00e9\u010dit z\u00e1va\u017en\u00e1 onemocn\u011bn\u00ed.3 Za\u010d\u00ednaj\u00ed se vyu\u017e\u00edvat jak ve sv\u011bt\u011b, tak i v \u010cesk\u00e9 republice. 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