{"id":6221,"date":"2020-07-14T19:54:38","date_gmt":"2020-07-14T16:54:38","guid":{"rendered":"http:\/\/blog.ulubat.org\/?p=6221"},"modified":"2020-07-14T19:54:41","modified_gmt":"2020-07-14T16:54:41","slug":"gen-terapisi-ve-sonen-umutlar","status":"publish","type":"post","link":"https:\/\/blog.ulubat.org\/index.php\/genel\/gen-terapisi-ve-sonen-umutlar\/","title":{"rendered":"GEN TERAP\u0130S\u0130 VE S\u00d6NEN UMUTLAR"},"content":{"rendered":"\n<p><strong>GEN TERAP\u0130S\u0130 NED\u0130R?<\/strong><\/p>\n\n\n\n<p>Gen terapisi, gitgide yayg\u0131nla\u015fan ve bir\u00e7ok ki\u015fide merak uyand\u0131ran bir ara\u015ft\u0131rma konusu haline geldi. DNA\u2019y\u0131 anlayabilmek ve ki\u015fiye fayda sa\u011flayacak \u015fekilde de\u011fi\u015ftirebilmek i\u00e7in bilmemiz gereken \u00e7ok fazla \u015fey var,\u00a0ancak bu ba\u015far\u0131ya ula\u015fabilmek i\u00e7in yap\u0131lan \u00e7al\u0131\u015fmalar bir\u00e7ok geli\u015fmeye imza at\u0131yor. Gen terapisinin \u00f6n\u00fcndeki engellerden biri, hastal\u0131klar\u0131n genetik temelinin karma\u015f\u0131kl\u0131\u011f\u0131d\u0131r (1). Bir\u00e7ok hastal\u0131k,\u00a0genetik ve \u00e7evresel fakt\u00f6rler ile kendini g\u00f6stermektedir. Buna ra\u011fmen hala her y\u0131l binlerce ki\u015fiyi etkileyen, kistik fibrozis ve orak h\u00fccre anemisi gibi tek gen hastal\u0131klar\u0131 da bulunmaktad\u0131r. DNA dizileme y\u00f6ntemleriyle elde edilmi\u015f olan bilgiler, bu hastal\u0131klar\u0131n tedavisinde kullan\u0131labilecek gen dizisi de\u011fi\u015fimlerinin de anla\u015f\u0131labilmesini kolayla\u015ft\u0131rm\u0131\u015ft\u0131r. Genom d\u00fczenleme y\u00f6ntemlerinin geli\u015ftirilip klinik d\u00fczeyde kullan\u0131labilmesi i\u00e7in,\u00a0bu y\u00f6ntemlerin do\u011fru diziyi hedefleyebilmesi, verimli i\u015fleyebilmesi ve hedef h\u00fccrelere\/dokulara ula\u015fabilmesi gereklidir. CRISPR-Cas9 gibi mekanizmalar\u0131n\u00a0do\u011fru h\u00fccrelerde do\u011fru dizileri do\u011fru \u015fekilde etkilediklerinden emin olundu\u011fu\u00a0zaman\u00a0ancak\u00a0bu mekanizmalar\u00a0g\u00fcvenilir bir \u015fekilde terapi haline getirilebilir.<\/p>\n\n\n\n<figure class=\"wp-block-image\"><img loading=\"lazy\" width=\"1024\" height=\"1024\" src=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1024x1024.png\" alt=\"\" class=\"wp-image-6222\" srcset=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1024x1024.png 1024w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-150x150.png 150w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-250x250.png 250w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-125x125.png 125w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-110x110.png 110w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-420x420.png 420w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-768x768.png 768w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption><a href=\"https:\/\/www.google.com\/url?sa=i&amp;url=https%3A%2F%2Fwww.docwirenews.com%2Fdocwire-pick%2Fhow-does-gene-therapy-work%2F&amp;psig=AOvVaw2J22LFl8C6Qgtb7AZcWJF7&amp;ust=1594659690619000&amp;source=images&amp;cd=vfe&amp;ved=0CA0QjhxqFwoTCOC3yreYyOoCFQAAAAAdAAAAABAD\">https:\/\/www.google.com\/url?sa=i&amp;url=https%3A%2F%2Fwww.docwirenews.com%2Fdocwire-pick%2Fhow-does-gene-therapy-work%2F&amp;psig=AOvVaw2J22LFl8C6Qgtb7AZcWJF7&amp;ust=1594659690619000&amp;source=images&amp;cd=vfe&amp;ved=0CA0QjhxqFwoTCOC3yreYyOoCFQAAAAAdAAAAABAD<\/a><\/figcaption><\/figure>\n\n\n\n<p>Genom d\u00fczenleme s\u00fcreci s\u0131ras\u0131nda,\u00a0farkl\u0131 \u015fekillerde istenmeyen de\u011fi\u015fiklikler olu\u015fabilir. \u00d6rne\u011fin incelenen DNA dizisindeki de\u011fi\u015fim, istenmeyen bir b\u00f6lgede ya\u015fanabilir veya enzim do\u011fru b\u00f6lgeye\u00a0ula\u015fsa bile\u00a0istenmeyen ve hastaya zarar verecek bir mutasyon yaratabilir. Bu yanl\u0131\u015flar \u00e7ok s\u0131k\u00a0g\u00f6r\u00fclmese de\u00a0baz\u0131lar\u0131\u00a0gen\u00a0terapisi\u00a0s\u0131ras\u0131nda hastada kanser olu\u015fumuna neden olabilecekleri i\u00e7in tehlikeli de olabilirler (1). Karolinska Enstit\u00fcs\u00fcnde yap\u0131lan bir ara\u015ft\u0131rma, CRISPR-Cas9 ile genomlar\u0131 de\u011fi\u015ftirilen h\u00fccrelerde p53 proteiniyle ilgili\u00a0sorunlar\u0131 ortaya \u00e7\u0131karm\u0131\u015ft\u0131r\u00a0(2). p53 proteini, CRISPR-Cas9 mekanizmas\u0131n\u0131n verimini azaltt\u0131\u011f\u0131 i\u00e7in hedef geni istenen \u015fekilde de\u011fi\u015ftirilmi\u015f h\u00fccreleri se\u00e7mek, i\u015flevsiz p53 proteinine sahip olan h\u00fccreleri se\u00e7mek anlam\u0131na da gelebilir (3). Dolay\u0131s\u0131yla bu h\u00fccrelerin kansere neden olabilme olas\u0131l\u0131\u011f\u0131 de de\u011ferlendirilmelidir.\u00a0\u00a0<\/p>\n\n\n\n<p><strong>JESSE GELSINGER<\/strong><\/p>\n\n\n\n<p>Jesse Gelsinger, OTCD (<em>ornithine transcarbamylase deficiency\/ornitin transkarbamilaz eksikli\u011fi<\/em>) denilen, ornitin transkarbamilaz enziminin eksikli\u011fiyle v\u00fccutta amonyak birikmesine neden olan metabolik bir bozuklu\u011fa sahipti (4). Erkeklerde\u00a0daha s\u0131k g\u00f6r\u00fclen bu hastal\u0131\u011f\u0131n en a\u011f\u0131r formu, yeni do\u011fanlarda\u00a0daha\u00a0yayg\u0131nd\u0131r (5). Jesse, hastal\u0131\u011f\u0131n\u0131n kendisine verdi\u011fi zarar\u0131n olabildi\u011fince azalabilmesi i\u00e7in beslenmesine \u00e7ok dikkat ediyordu ve bu \u015fekilde de hastal\u0131\u011f\u0131yla ba\u015f etmeyi ba\u015farabilmi\u015fti (6).<\/p>\n\n\n\n<figure class=\"wp-block-image\"><img loading=\"lazy\" width=\"1024\" height=\"1024\" src=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-1024x1024.png\" alt=\"\" class=\"wp-image-6223\" srcset=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-1024x1024.png 1024w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-150x150.png 150w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-250x250.png 250w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-125x125.png 125w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-110x110.png 110w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-1-768x768.png 768w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption>Jesse Gelsinger.\u00a0<a href=\"https:\/\/www.google.com\/url?sa=i&amp;url=https%3A%2F%2Fen.wikipedia.org%2Fwiki%2FJesse_Gelsinger&amp;psig=AOvVaw2b2hMVg_CXQOwiN7MMrpDj&amp;ust=1594659810228000&amp;source=images&amp;cd=vfe&amp;ved=0CA4QjhxqFwoTCIDk8e6YyOoCFQAAAAAdAAAAABAD\">https:\/\/www.google.com\/url?sa=i&amp;url=https%3A%2F%2Fen.wikipedia.org%2Fwiki%2FJesse_Gelsinger&amp;psig=AOvVaw2b2hMVg_CXQOwiN7MMrpDj&amp;ust=1594659810228000&amp;source=images&amp;cd=vfe&amp;ved=0CA4QjhxqFwoTCIDk8e6YyOoCFQAAAAAdAAAAABAD<\/a><\/figcaption><\/figure>\n\n\n\n<p>G\u00fcnde neredeyse 50 tane hap alarak bu hastal\u0131kla ya\u015fam\u0131n\u0131 s\u00fcrd\u00fcrmeye\u00a0\u00e7al\u0131\u015fan\u00a0Jesse, Pensilvanya \u00dcniversitesindeki ara\u015ft\u0131rmac\u0131lar\u0131n \u00e7al\u0131\u015fmalar\u0131ndan haberdar olur (4). Kendi hayat kalitesinde b\u00fcy\u00fck bir geli\u015fme g\u00f6r\u00fclmesi hedeflenmese bile gelecekte bu hastal\u0131\u011f\u0131n tedavisinde kullan\u0131labilecek gen terapisinin geli\u015ftirilmesine katk\u0131da bulunmak istiyordur; g\u00f6n\u00fcll\u00fc olur ve bu ama\u00e7la da 1999 y\u0131l\u0131 Eyl\u00fcl ay\u0131nda ara\u015ft\u0131rman\u0131n y\u00fcr\u00fct\u00fcld\u00fc\u011f\u00fc yere gider. Bu ara\u015ft\u0131rmac\u0131lar, gen terapisi ile OTCD\u2019li farelerde OTC enzimini tekrar i\u015flevli hale getirerek onlar\u0131n ya\u015famlar\u0131n\u0131 uzatmay\u0131 ba\u015farm\u0131\u015flard\u0131 (4). Jesse\u2019den \u00f6nce bu klinik ara\u015ft\u0131rmaya kat\u0131lan\u00a0di\u011fer\u00a0ki\u015filerin so\u011fuk alg\u0131nl\u0131\u011f\u0131na benzer belirtiler ge\u00e7irdikleri kaydedilmi\u015fti, dolay\u0131s\u0131yla kimse Jesse\u2019ninki kadar a\u011f\u0131r bir gidi\u015fat beklemiyordu. Jesse terap\u00f6tik Adenovir\u00fcs vekt\u00f6r\u00fcn\u00fc ald\u0131ktan \u00e7ok k\u0131sa bir s\u00fcre i\u00e7inde akci\u011fer, karaci\u011fer ve b\u00f6breklerinde tehlikeli problemler ya\u015fad\u0131. Tedaviden d\u00f6rt g\u00fcn sonra da hayat\u0131n\u0131 kaybetti. Jesse\u2019nin h\u0131zl\u0131 ve beklenmedik\u00a0\u00f6l\u00fcm\u00fc, toplumun ve baz\u0131 proje destekleyicilerinin de gen terapisine bak\u0131\u015f a\u00e7\u0131s\u0131n\u0131 olumsuz \u015fekilde etkiledi. CRISPR gibi y\u00f6ntemlerin geli\u015ftirilmesiyle belki de daha g\u00fcvenli olabilecek gen terapileri\u00a0\u00fczerine\u00a0\u00e7al\u0131\u015f\u0131l\u0131yor ancak\u00a0hala\u00a0bu terapilerin olas\u0131 olumsuz etkileri hakk\u0131nda tart\u0131\u015fmalar da devam ediyor.\u00a0<\/p>\n\n\n\n<figure class=\"wp-block-image\"><img loading=\"lazy\" width=\"1024\" height=\"1024\" src=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-1024x1024.png\" alt=\"\" class=\"wp-image-6224\" srcset=\"https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-1024x1024.png 1024w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-150x150.png 150w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-250x250.png 250w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-125x125.png 125w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-110x110.png 110w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-420x420.png 420w, https:\/\/blog.ulubat.org\/wp-content\/uploads\/2020\/07\/image-2-768x768.png 768w\" sizes=\"(max-width: 1024px) 100vw, 1024px\" \/><figcaption>Adenovir\u00fcslerin gen terapisinde genel olarak kullan\u0131m \u015fekli. https:\/\/www.sciencedirect.com\/science\/article\/pii\/S2352304217300272 <br><\/figcaption><\/figure>\n\n\n\n<p>Jesse\u2019nin neden kaybedildi\u011fi ara\u015ft\u0131r\u0131l\u0131p anla\u015f\u0131lmaya \u00e7al\u0131\u015f\u0131l\u0131rken de bu ara\u015ft\u0131rman\u0131n, Jesse kat\u0131lmadan \u00f6nceki d\u00f6nemde y\u00fcr\u00fct\u00fclme \u015fekilleri ve bulgular\u0131 hakk\u0131nda da yeni bilgiler ortaya \u00e7\u0131kt\u0131. Jesse\u2019nin denemelere kat\u0131lmadan \u00f6nce di\u011fer hastalar\u0131n ya\u015fam\u0131\u015f olduklar\u0131 yan etkiler hakk\u0131nda yeterince bilgilendirilmemesi; denemelerden \u00f6nce yap\u0131lan karaci\u011fer i\u015flev testlerinde Jesse\u2019nin karaci\u011ferinin d\u00fc\u015f\u00fck performans g\u00f6stermesine ra\u011fmen denemelere devam\u00a0edilmesi, bunlar aras\u0131nda say\u0131labilir (3). Jesse\u2019nin kaybedilmesinden hemen sonra elde edilen bilgiler, ilk ba\u015fta Jesse\u2019nin g\u00f6sterdi\u011fi \u00f6l\u00fcmc\u00fcl tepkinin rastgele ve \u00f6ng\u00f6r\u00fclemez oldu\u011funu d\u00fc\u015f\u00fcnd\u00fcrm\u00fc\u015ft\u00fc ancak deneme verilerinin daha detayl\u0131 incelenmesi, farkl\u0131 \u015feylerin de ger\u00e7ekle\u015fmi\u015f olabilece\u011fini ortaya koydu (6). \u00d6rne\u011fin \u00e7al\u0131\u015fmada baz\u0131 deney hayvanlar\u0131n\u0131n ve ba\u015fka hastalar\u0131n da ciddi yan etkiler g\u00f6sterdi\u011finin, Jesse de dahil olmak \u00fczere denemelere kat\u0131lan di\u011fer\u00a0hastalara, olu\u015fabilecek yan etkilerin do\u011fru\u00a0\u015fekilde s\u00f6ylenmemesi etik tart\u0131\u015fmalar yaratt\u0131. Jesse\u2019nin ailesi, o tedavi i\u00e7in g\u00f6n\u00fcll\u00fc olurken bu durumun onlara a\u00e7\u0131klanmad\u0131\u011f\u0131n\u0131, dolay\u0131s\u0131yla Jesse\u2019nin yeterince bilgi sahibi olmayarak bu i\u015fin i\u00e7ine girdi\u011fini s\u00f6ylediler (6). T\u00fcm bunlara ek olarak, Pensilvanya \u00dcniversitesi \u0130nsan Gen Terapi Enstit\u00fcs\u00fc\u00a0Ba\u015fkan\u0131 James Wilson\u2019\u0131n yapt\u0131\u011f\u0131 \u00e7al\u0131\u015fmada bir \u00e7\u0131kar \u00e7at\u0131\u015fmas\u0131 i\u00e7inde oldu\u011fu da d\u00fc\u015f\u00fcn\u00fcld\u00fc (3). Haberlere g\u00f6re\u00a0Wilson\u2019\u0131n\u00a0Adenovir\u00fcslerle yapt\u0131\u011f\u0131 \u00e7al\u0131\u015fmalar\u0131n olumlu sonu\u00e7lanmas\u0131ndan\u00a0Wilson finansal olarak gelir elde edecekti\u00a0(6). Yani bu durumda klinik \u00e7al\u0131\u015fmalar\u0131nda verdi\u011fi kararlar\u00a0da\u00a0elde edece\u011fi fayday\u0131 etkileyecekti. \u0130ddialar\u0131 reddeden Wilson, Jesse\u2019de g\u00f6r\u00fclen durumun \u00f6ng\u00f6r\u00fclemeyecek bir \u015fey oldu\u011funu ve maddi kazanc\u0131n verdi\u011fi kararlar\u0131 etkilemedi\u011fini s\u00f6ylemi\u015fti (3). Dolay\u0131s\u0131yla Jesse\u2019nin sa\u011fl\u0131\u011f\u0131n\u0131n bu kadar h\u0131zl\u0131 bir \u015fekilde k\u00f6t\u00fcye gitmesi ve ya\u015fanan durum hem gen terapisi hem de bu \u00e7al\u0131\u015fma hakk\u0131nda ak\u0131llarda bir\u00e7ok soruya\u00a0neden olmu\u015ftu.<\/p>\n\n\n\n<p><strong>TECR\u00dcBELER VE GEN TERAP\u0130S\u0130N\u0130N GELECE\u011e\u0130<\/strong><\/p>\n\n\n\n<p>Arkas\u0131ndaki ger\u00e7ekler ne olursa olsun, Jesse\u2019nin gen terapisi denemelerinde&nbsp;hayat\u0131n\u0131 kaybetmesi&nbsp;bu alan\u0131n bir\u00e7ok a\u00e7\u0131dan yeniden g\u00f6zden ge\u00e7irilmesini ve bir\u00e7ok s\u0131n\u0131rland\u0131r\u0131lman\u0131n y\u00fcr\u00fcrl\u00fc\u011fe girmesini&nbsp;de&nbsp;beraberinde getirdi. \u00d6rne\u011fin Amerika\u2019da FDA (Food and Drug Administration\/Amerikan G\u0131da ve \u0130la\u00e7 Dairesi) ve NIH (National Institute of Health\/Ulusal Sa\u011fl\u0131k Enstit\u00fcs\u00fc) kurumlar\u0131, daha \u00f6nceki gen terapi \u00e7al\u0131\u015fmalar\u0131n\u0131n verilerini incelediklerinde,&nbsp;Jesse\u2019nin \u00f6l\u00fcm\u00fcnden \u00f6nceki yedi y\u0131l i\u00e7erisinde denemelere kat\u0131lan 691 g\u00f6n\u00fcll\u00fcn\u00fcn hayat\u0131n\u0131 kaybetti\u011fini veya hastaland\u0131\u011f\u0131n\u0131, ancak bunlar i\u00e7erisinden sadece 39&nbsp;ki\u015finin&nbsp;k\u0131sa s\u00fcre i\u00e7inde kay\u0131tlara ge\u00e7irildi\u011fini buldu (3).&nbsp;<\/p>\n\n\n\n<p>T\u00fcm bunlar d\u00fc\u015f\u00fcn\u00fcld\u00fc\u011f\u00fcnde, gen terapisi hakk\u0131nda umutsuzlu\u011fa kap\u0131lmak m\u00fcmk\u00fcn olabilir ama\u00a0asl\u0131nda\u00a0durum g\u00f6r\u00fcnd\u00fc\u011f\u00fcnden\u00a0daha ayd\u0131nl\u0131k. Gen terapisi, Jesse\u2019den \u00f6nce ve sonra da denenmi\u015f ve birka\u00e7 durumda ba\u015far\u0131l\u0131 olmu\u015f veya tedavinin gelece\u011fi hakk\u0131nda de\u011ferli bilgiler edinilmesini sa\u011flam\u0131\u015ft\u0131r. AK\u0130Y (A\u011f\u0131r Kombine \u0130mm\u00fcn Yetmezlik), gen terapisi ile tedavi edilmeye \u00e7al\u0131\u015f\u0131lan ilk hastal\u0131klardand\u0131r. Fransa\u2019da 2002 y\u0131l\u0131nda 10 \u00e7ocukla yap\u0131lan klinik denemeler, ilk ba\u015fta hepsinde ba\u015far\u0131l\u0131 olmu\u015ftur ancak ilerleyen y\u0131llarda \u00e7ocuklardan baz\u0131lar\u0131nda l\u00f6semi geli\u015fmesi ve bir tanesinin de hayat\u0131n\u0131 kaybetmesiyle sonland\u0131r\u0131lm\u0131\u015ft\u0131r (7). Bu \u00fcz\u00fcc\u00fc kay\u0131plara ra\u011fmen denemelere kat\u0131lan di\u011fer \u00e7ocuklar tedaviden fayda g\u00f6rm\u00fc\u015ft\u00fcr (8). Adenozin deaminaz (ADA) eksikli\u011finden kaynaklanan genetik bir hastal\u0131\u011fa sahip\u00a0olanAshanti Desilva,\u00a0di\u011fer tedavi t\u00fcrlerine cevap vermemeye ba\u015flam\u0131\u015fken gen terapisiyle iyile\u015ftirilmi\u015ftir (9). Onun\u00a0gidi\u015fat\u0131 ve\u00a0hayat\u0131na \u201cnormal biri\u201d gibi devam edebilmesi bu denemelere ba\u015fka \u00e7ocuklarla da devam edilmesini, ba\u015fka bir k\u0131z \u00e7ocu\u011funun daha tedavi edilebilmesini sa\u011flam\u0131\u015ft\u0131r. Bu g\u00fczel geli\u015fmelere ek olarak ba\u015fka ba\u015far\u0131l\u0131 gen terapisi \u00f6rnekleri de bulunabilir:\u00a0Kal\u0131tsal k\u00f6rl\u00fck ve\u00a0hemofili\u00a0(7) gibi hastal\u0131klar\u0131n gen terapisi ile tedavisi i\u00e7in de sevindirici sonu\u00e7lar elde edilmi\u015ftir. Bir\u00e7ok hastal\u0131k i\u00e7in de gen terapisinin potansiyeli ve y\u00f6ntemleri ara\u015ft\u0131r\u0131lmaktad\u0131r. Gen terapisi; bilimin geldi\u011fi bu \u00e7a\u011fda imkans\u0131z bir durum olmak yerine, kullan\u0131m stratejileri belirlenmesi, \u00e7ok dikkatli bir \u015fekilde uygulanmas\u0131 ve\u00a0do\u011fru\u00a0noktaya ula\u015f\u0131labilmesi i\u00e7in de hakk\u0131nda ara\u015ft\u0131rma y\u00fcr\u00fct\u00fclmesi gereken, bir\u00e7ok insan\u0131n hayat\u0131n\u0131 de\u011fi\u015ftirebilecek bir y\u00f6ntemdir. Dolay\u0131s\u0131yla bilim insanlar\u0131n\u0131n bu konuda \u00e7al\u0131\u015f\u0131rken yava\u015f ve tedbirli ilerlemeleri gerekti\u011fi d\u00fc\u015f\u00fcn\u00fclmektedir (10).<\/p>\n\n\n\n<p><strong>KAYNAK\u00c7A<\/strong><\/p>\n\n\n\n<ol><li>Doudna, J. (2020). The promise and challenge of therapeutic genome editing.\u00a0<em>Nature<\/em>, 578.<\/li><li>Haapaniemi, E., Botla, S., Persson, J.\u00a0<em>et al.<\/em>\u00a0(2018). CRISPR\u2013Cas9 genome editing induces a p53-mediated DNA damage response.\u00a0<em>Nat Med\u00a0<\/em><strong>24,\u00a0<\/strong>927\u2013930.\u00a0<a href=\"https:\/\/doi.org\/10.1038\/s41591-018-0049-z\">https:\/\/doi.org\/10.1038\/s41591-018-0049-z<\/a><\/li><li>Karolinska Institutet. (2018, June 11). Genome-editing tool could increase cancer risk.\u00a0<em>ScienceDaily<\/em>. Retrieved July 12, 2020 from\u00a0<a href=\"http:\/\/www.sciencedaily.com\/releases\/2018\/06\/180611133816.htm\">www.sciencedaily.com\/releases\/2018\/06\/180611133816.htm<\/a>. Eri\u015fim tarihi: 12 Temmuz 2020.<\/li><li>Science History Institute, \u2018The Death of Jesse Gelsinger, 20 Years Later.\u2019 4 Haziran 2019.\u00a0<a href=\"https:\/\/www.sciencehistory.org\/distillations\/the-death-of-jesse-gelsinger-20-years-later\">https:\/\/www.sciencehistory.org\/distillations\/the-death-of-jesse-gelsinger-20-years-later<\/a>. Eri\u015fim tarihi: 11 Temmuz 2020.<\/li><li>Genetics Home Reference. (2020). Ornithine transcarbamylase deficiency.\u00a0<a href=\"https:\/\/ghr.nlm.nih.gov\/condition\/ornithine-transcarbamylase-deficiency\">https:\/\/ghr.nlm.nih.gov\/condition\/ornithine-transcarbamylase-deficiency<\/a>. Eri\u015fim tarihi: 12 Temmuz 2020.<\/li><li>NYU High School Bioethics. \u2018The Case of Jesse Gelsinger\u2019.\u00a0<a href=\"https:\/\/med.nyu.edu\/highschoolbioethics\/briefs\/jesse-gelsinger\">https:\/\/med.nyu.edu\/highschoolbioethics\/briefs\/jesse-gelsinger<\/a>. Eri\u015fim tarihi: 14 Temmuz 2020.<\/li><li>University of Utah Genetic Science Learning Center. \u2018Gene Therapy Successes\u2019.\u00a0<a href=\"https:\/\/learn.genetics.utah.edu\/content\/genetherapy\/success\/\">https:\/\/learn.genetics.utah.edu\/content\/genetherapy\/success\/<\/a>. Eri\u015fim tarihi: 14 Temmuz 2020.<\/li><li>Edelstein, M., Abedi, M., &amp; Wixon, J. (2007). Gene therapy clinical trials worldwide to 2007\u2014an update.\u00a0<em>The Journal Of Gene Medicine<\/em>,\u00a0<em>9<\/em>(10), 833-842. doi: 10.1002\/jgm.1100\u00a0<\/li><li>Premier Research, Juliet Moritz. (November 14, 2018). The Four-Year-Old That Changed History\u2019.\u00a0<a href=\"https:\/\/premier-research.com\/blog\/perspectives-ashanthi-desilva-gene-therapy-trial\/\">https:\/\/premier-research.com\/blog\/perspectives-ashanthi-desilva-gene-therapy-trial\/<\/a>. Eri\u015fim tarihi: 14 Temmuz 2020.<\/li><li>McCain, J. (2005). The future of gene therapy.\u00a0<em>Biotechnology healthcare<\/em>,\u00a0<em>2<\/em>(3), 52\u201360.<\/li><\/ol>\n","protected":false},"excerpt":{"rendered":"<p>GEN TERAP\u0130S\u0130 NED\u0130R? Gen terapisi, gitgide yayg\u0131nla\u015fan ve bir\u00e7ok ki\u015fide merak uyand\u0131ran bir ara\u015ft\u0131rma konusu haline geldi. DNA\u2019y\u0131 anlayabilmek ve<\/p>\n","protected":false},"author":273,"featured_media":6225,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[1],"tags":[95,808,149,158,115,810,807,809],"acf":[],"views":1261,"_links":{"self":[{"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/posts\/6221"}],"collection":[{"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/users\/273"}],"replies":[{"embeddable":true,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/comments?post=6221"}],"version-history":[{"count":1,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/posts\/6221\/revisions"}],"predecessor-version":[{"id":6226,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/posts\/6221\/revisions\/6226"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/media\/6225"}],"wp:attachment":[{"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/media?parent=6221"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/categories?post=6221"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/blog.ulubat.org\/index.php\/wp-json\/wp\/v2\/tags?post=6221"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}