Ballanqaadyada Daaweynta Cusub
Ilaa hadda, ma jirto daawo loogu talagalay cystic fibrosis (CF), laakiin cilmi-baarayaashu si adag ayey u shaqeynayaan si ay u helaan mid. Cilmi-baadhayaashu way ku dhow yihiin weligoodba. Dhowrkii sano ee la soo dhaafay, jir ballaaran oo cilmi baaris ah ayaa sababay horumarinta daroogooyin cusub iyo daaweynyo si weyn u hagaajiyay rajada nolosha iyo tayada nolosha. Dhamaan kuwan ayaa albaabada u furay baaritaan cusub oo keeni kara daaweyn.
Kuwa soo socda ayaa ah qaar ka mid ah daaweynta suurtagalka ah ee la baranayo.
Gene Therapy
Sanadkii 1989-kii, gene-ka mas'uul ka ah inuu keeno cystic fibrosis ayaa la ogaaday: hiddaha CFIT . Natiijadani waxay ahayd mid xiiso u leh bulshada CF. Dad badan ayaa aaminsan in helitaanku uu horseedi doono daaweyn hiddesid hiddesid.
Nasiib darro, taasi wali ma dhicin, laakiin ma ahan sabab la'aan. Daraasado farabadan ayaa la sameeyay si loo isku dayiyo loona saxo ciladda hiddaha, laakiin midkoodna ma guuleysan. Dhibaatada ugu weyn ee daaweynta hidda-wadda ilaa iyo haatanba waxay raadineysaa farsamo si wax ku ool ah u hirta hannaanka saxda ah ee unugyada.
Waxaa laga yaabaa inay weli rajo ka qabaan daweynta hidda-wadaha, inkastoo. Bishii Luulyo 2009, koox cilmi baarayaal ah oo ka tirsan Jaamacadda North Carolina ee Chapel Hill waxay natiijooyin aad ufiican u adeegsadeen fayras caadi ah oo hargab ah sida vektor ah si loogu wareejiyo hiddaha shaybaarka shaybarka ee unugyada sanbabada. Kooxda cilmi baarista ayaa hadda ka shaqeyneysa habka lagu yareeyo fayruuska qabow si daaweynta loogu tijaabiyo dadka qaba cystic fibrosis.
VX-770
VX-770 waa daawo lagu baaro dawooyinka Vertex ee dadka qaba cystic fibrosis kuwaas oo leh ugu yaraan hal nuqul oo ah isbeddelka G551D. Daroogada ayaa dhab ahaantii awood u leh inay bartilmaameedsato cilladda CFIT waxayna dib u soo celineysaa awooda ay u leedahay in ay furto jimicsiga chloride, sidaas darteed u oggolaanaya cusbada in ay ku dhex socoto oo ka baxdo unugyada si sax ah.
Si ka duwan sida loo daweeyo hiddaha, VX-770 ma badali doonto hiddada cilladaysan. Taas bedelkeeda, haddii uu guuleysto, VX-770 waxay ku hagaajin doontaa dhibaatada hiddaha hadda jira.
VX-809
VX-809 waa daroogo kale oo lagu baaro daawooyinka Vertex ee dadka haysta laba nuqul oo ka mid ah mutacal ΔF508-CFTR. Waxay u egtahay VX-770 in laga yaabo in ay kudhiibto oo ay ku hesho unugyada si haboon, laakiin waxay si ka duwan u shaqeysaa. Haddii ay ka shaqeyso habka ay cilmi-baarayaashu rajeynayaan in ay u baahan tahay, VX-809 waxay furi doontaa kanaalka chloride iyada oo u gudbinaysa borotiinka CFIT ilaa meesheeda ku habboon mashiinka unugyada hawada.
Miglustat
Miglustat waa daawo ay soo saartay Pharmaceutical Pharmaceuticals kaas oo horeyba loo isticmaalay si loogu daaweeyo xaalado kale, hase yeeshee waxaa haatan loo baaray si loogu isticmaalo dadka qaba cystic fibrosis kuwaas oo haysta laba nuqul oo ah mutarjuminta ΔF508-CFTR. Daraasadani waa yar tahay (waxay ka kooban tahay 15 qof oo keliya), laakiin ilaa hadda natiijooyinka ayaa la rajeynayay. Miglustat waxay awoodday inay dib u celiso CFST cillad oo dib u soo celiso dhaqdhaqaaqa caadiga ah unugyada.
Ataluren
Ataluren, oo mar la yiraahdo PTC124, waxaa lagu baranayaa PTC Therapeutics sida daaweynta suurtagalka ah ee loogu talagalay dadka qaba CF oo leh miyir-qabad aan caadi ahayn. In isku-dhafnaan micnaheedu yahay, qayb ka mid ah code "gibberish" ayaa u muuqda iyada oo la raacayo xeerka caadiga ah ee hiddaha CFIT.
Xeerka sirta ah wuxuu u dhaqmaa sida calaamad joojin, ka horjoogsanaya unugyada akhriska wixii koodh ah oo dhaca kadib. Ataluren waxay awoodi kartaa in ay saxdo dhibaatadan adigoo ka caawinaya unugyada in ay iska indho tiraan calaamadda joogsiga oo ay sii wadaan akhrinta koodka ka dambeeya, kadibna dib u soo celinta shaqooyinka caadiga ah ee unugyada.
> Ilo:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "Cudurka Epirelial Respiratory System ee Kronik Miisaanka lagu Daweeyo Miglustat wuxuu aqbalaa" Non-CF Like Phenotype ". Wargayska American ee Neefsashada Neefsashada iyo Bayoolajiyada Molecular . Agoosto 2009.
> Foundation Foundation Fibrosis. Juun 2009. Gaadiidka Horumarinta Daroogada. 24 Luulyo 2009.
> Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, iyo al. 2009 "CFTR Delivery ilaa 25% oo ah Unugyada Epithelial Nooca Dib u soo Celinta Heerarka caadiga ah ee Gaadiidka Mucus ee loo yaqaan" Epibelium-ka Fibrosis "ee" Human Fibrosis ". PLO Biol 7 (7): e1000155. doi: 10.1371 / journal.pbio.1000155. 24 Luulyo 2009.