{"id":31691,"date":"2019-01-07T01:00:32","date_gmt":"2019-01-06T22:00:32","guid":{"rendered":"https:\/\/bilimvegelecek.com.tr\/?p=31691"},"modified":"2019-01-06T20:10:06","modified_gmt":"2019-01-06T17:10:06","slug":"2018de-bilimde-ders-kitaplarini-degistirecek-neler-oldu-6","status":"publish","type":"post","link":"https:\/\/bilimvegelecek.com.tr\/index.php\/2019\/01\/07\/2018de-bilimde-ders-kitaplarini-degistirecek-neler-oldu-6","title":{"rendered":"2018\u2019de bilimde ders kitaplar\u0131n\u0131 de\u011fi\u015ftirecek neler oldu?"},"content":{"rendered":"

7) Patisiran<\/b> pahal\u0131ya patlad\u0131!
\nRNAi tabanl\u0131 ilk ila\u00e7 onay ald\u0131…<\/b><\/p>\n

20 y\u0131ld\u0131r devam eden hummal\u0131 bir \u00e7aba, bol s\u0131f\u0131rl\u0131 yat\u0131r\u0131mlar, Nobel \u00f6d\u00fcl\u00fcn\u00fc getirecek kadar b\u00fcy\u00fck bir heyecan, ard\u0131ndan gelen hayal k\u0131r\u0131kl\u0131\u011f\u0131, h\u00fcsran ve vazge\u00e7i\u015f\u2026 RNAi (RNA interferans) denen molek\u00fcl\u00fcn maceral\u0131 \u00f6yk\u00fcs\u00fc 10 A\u011fustos 2018\u2019de nihayet mutlu sonla bitti: Amerikan \u0130la\u00e7 ve G\u0131da Dairesi (FDA) RNAi tabanl\u0131 ilk ilaca onay verdi\u011fini duyurdu.<\/span><\/p>\n

RNAi hayvan ve bitki h\u00fccrelerinde do\u011fal olarak \u00fcretilen bir vir\u00fcs-savar mekanizma asl\u0131nda. Ayn\u0131 zamanda hasarl\u0131 protein \u00fcreten genlerin \u201csusturulmas\u0131n\u0131\u201d da sa\u011flayan bir kontrol sistemi. Yakla\u015f\u0131k 20 y\u0131l \u00f6nce \u00e7\u00f6z\u00fclen ve k\u00e2\u015fifleri Andrew Fire (Stanford \u00dcniversitesi) ve Craig Mello\u2019ya (Massachusettes \u00dcniversitesi) Nobel \u00f6d\u00fcl\u00fc kazand\u0131ran bu molek\u00fcler mekanizma uzun s\u00fcredir ila\u00e7 formuna sokulmaya \u00e7al\u0131\u015f\u0131l\u0131yordu. \u0130\u015fleyi\u015f ilkesi ayn\u0131 kalacakt\u0131; yani hasarl\u0131 proteinin \u00fcretimine arac\u0131l\u0131k eden ve ilgili genin dizisini ta\u015f\u0131yan arac\u0131 mRNA molek\u00fcl\u00fc (genlerden protein elde edilmesi a\u015famas\u0131nda rol alan arac\u0131 RNA molek\u00fcl\u00fc), bu diziye kar\u015f\u0131l\u0131k gelen, d\u0131\u015far\u0131dan ila\u00e7 formunda verilmi\u015f \u00f6zel RNA molek\u00fclleri arac\u0131l\u0131\u011f\u0131yla par\u00e7alanacak, b\u00f6ylece geride hastal\u0131\u011fa yol a\u00e7an bir unsur kalmam\u0131\u015f olacakt\u0131. Gel g\u00f6r ki bu \u00f6zel tasar\u0131m RNA molek\u00fcllerini hedef dokuya bozulmadan ve metabolizman\u0131n h\u0131\u015fm\u0131na u\u011framadan yollamak b\u00fcy\u00fck sorun oldu. \u00d6yle ki bir noktada umudunu kesen Roche, Merck, Abbott gibi ila\u00e7 devleri ara\u015ft\u0131rma programlar\u0131n\u0131 sonland\u0131rmaya, yat\u0131r\u0131mc\u0131lar el ayak \u00e7ekmeye ba\u015flad\u0131lar.\u00a0<\/span><\/p>\n

\"\"
Patisiran\u2019\u0131n bir kutusu hazine de\u011ferinde<\/figcaption><\/figure>\n

Ta ki FDA\u2019n\u0131n onay verdi\u011fi ilac\u0131n \u00fcreticisi Alnylam, molek\u00fclleri nanopartik\u00fcller i\u00e7inde muhafazaya al\u0131p ilgili organa sa\u011f salim g\u00f6ndermeyi ba\u015farana kadar. Gerisi \u00e7orap s\u00f6k\u00fc\u011f\u00fc gibi geldi. Molek\u00fcller karaci\u011ferde toplan\u0131nca ara\u015ft\u0131rmac\u0131lar bu organda \u00fcretilen hasarl\u0131 bir proteinle ili\u015fkili bir hastal\u0131\u011f\u0131 hedef olarak se\u00e7me karar\u0131 ald\u0131lar. B\u00f6ylece d\u00fcnyada yakla\u015f\u0131k 50.000 ki\u015fiyi etkileyen, nadir bir kal\u0131t\u0131msal hastal\u0131k olan transtiretin amiloidoz hastalar\u0131na g\u00fcn do\u011fdu. Sinir sistemi hasar g\u00f6ren bu hastalara uygulanan RNAi tedavisi sayesinde hasarl\u0131 transtiretin proteinlerine ait mRNA\u2019lar par\u00e7aland\u0131, dolay\u0131s\u0131yla hasarl\u0131 protein de yok olmu\u015f ve hastalardaki bu proteine ba\u011fl\u0131 harabiyet ciddi \u00f6l\u00e7\u00fcde gerilemi\u015f oldu. Ard\u0131ndan ver elini FDA; \u00f6nce Avrupa\u2019da, ard\u0131ndan ABD\u2019de \u201cpatisiran\u201d sat\u0131\u015f\u0131 onayland\u0131.<\/span><\/p>\n

Burada as\u0131l mesele mevcut ilac\u0131n hedefindeki hastal\u0131k de\u011fil, bu mekanizmaya dayal\u0131 ila\u00e7lar\u0131n yak\u0131n gelecekte vaat etti\u011fi t\u0131bbi devrim elbette. Yani s\u0131rada \u00e7ok daha yayg\u0131n ve can al\u0131c\u0131 hastal\u0131klar da var (mesela hemofili). Fakat yine de fikir vermesi a\u00e7\u0131s\u0131ndan belirtmeden ge\u00e7meyelim. Alnylam\u2019\u0131n RNAi tabanl\u0131 patisiran\u2019\u0131n\u0131n fiyat\u0131 350.000$ civar\u0131nda, mevcut kurla 2 milyon TL\u2019ye yak\u0131n. G\u00f6r\u00fcnen o ki ilac\u0131 elimize alabilmek i\u00e7in daha \u00e7ok bekleyece\u011fiz\u2026<\/span><\/p>\n

8) Bu kedi\u0307ler i\u0307flah olmaz!
\nDi\u011fer evcil hayvanlar\u0131n tersine, kediler giderek b\u00fcy\u00fcyor…<\/b><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

7) Patisiran pahal\u0131ya patlad\u0131! RNAi tabanl\u0131 ilk ila\u00e7 onay ald\u0131… 20 y\u0131ld\u0131r devam eden hummal\u0131 bir \u00e7aba, bol s\u0131f\u0131rl\u0131 yat\u0131r\u0131mlar, Nobel \u00f6d\u00fcl\u00fcn\u00fc getirecek kadar b\u00fcy\u00fck bir heyecan, ard\u0131ndan gelen hayal k\u0131r\u0131kl\u0131\u011f\u0131, h\u00fcsran ve vazge\u00e7i\u015f\u2026 RNAi (RNA interferans) denen molek\u00fcl\u00fcn maceral\u0131 \u00f6yk\u00fcs\u00fc 10 A\u011fustos 2018\u2019de nihayet mutlu sonla bitti: Amerikan \u0130la\u00e7 ve G\u0131da Dairesi (FDA) RNAi […]<\/p>\n","protected":false},"author":184,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[231,33],"tags":[4410,4408,4407],"class_list":["post-31691","post","type-post","status-publish","format-standard","hentry","category-molekuler-biyoloji-ve-genetik","category-saglik-bilimleri","tag-amerikan-ilac-ve-gida-dairesi-fda","tag-rna-mudahalesi","tag-rnai-rna-interferans"],"acf":[],"aioseo_notices":[],"aioseo_head":"\n\t\t\n\t\n\t\n\t\n\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t