Uthembisa iNyango entsha
Okwangoku, akukho nonyango ye- cystic fibrosis (CF), kodwa abaphandi basebenza nzima ukufumana enye. Iingcali zenzululwazi zisondele ngakumbi kunoko ngaphambili. Ngaphezulu kweminyaka emibini edluleyo, uphando olongezelelekileyo luye lwakhokelela ekuphuhlisweni kwamachiza amatsha kunye nokunyanga okuphuculwe ngokugqithiseleyo kokulindela ubomi kunye nomgangatho wobomi. Konke oku kuye kwavula iingcango zophando olutsha olukhokelela ekunyangeni.
Ezi zilandelayo ziphi ezinye zonyango ezinokuthi zifundwe.
LweTherapy
Ngo-1989, i-gene egxile ekubangela i-cystic fibrosis yafunyanwa: i- CFTR gene . Ukufumanisa oku kwavuya kummandla weCF. Abaninzi babekholelwa ukuba ukufumaneka bekuya kubangela unyango ngokusebenzisa unyango lwemizimba.
Ngelishwa, oko akukwenzekanga okwamanje, kodwa kungekhona ukungabi nako kokuzama. Izifundo ezininzi zenzelwe ukuzama ukulungisa i-defect genetic but none of them has been successful. Ingxaki enkulu kwi-gene therapy kuze kube ngoku ifunyenwe i-vector engakwazi ukuphatha kakuhle i- gene gene .
Kukho isenokuba nethemba le-gene, kodwa. NgoJulayi 2009, iqela labaphandi kwiYunivesithi yaseNorth Carolina kwiChapel Hill yayineempembelelo ezilungileyo kakhulu ngokusebenzisa intsholongwane efana nengqungquthela yokudlulisa i-gene kwiisampuli zebhubhoratri zamathambo emiphunga. Iqela lophando ngoku isebenza ngendlela yokunciphisa intsholongwane yebandayo ukuze unyango luhlolwe kubantu abane-cystic fibrosis.
VX-770
I-VX-770 yiyilisi evivinywa yi-Vertex Pharmaceuticals kubantu abane-cystic fibrosis abane-ubuncinane ikopi enye ye-G551D. Isilwanyana sinokwazi ukujolisa i-defect kwi-CFTR gene kunye nokubuyisela amandla ayo okuvula iziteshi ze-chloride, ngaloo ndlela uvumela ukuba ityuwa igele kwaye iphume kwiseli ngokufanelekileyo.
Ngokungafani nonyango lwe-gene, i-VX-770 ayiyi kuthatha indawo yesini esichasayo. Kunoko, ukuba uphumelele, i-VX-770 iya kulungisa ingxaki kwi-gene ekhoyo.
VX-809
I-VX-809 yinye imichiza ehlolwe yi-Vertex Pharmaceuticals kubantu abaneikopi ezimbini ze-ΔF508-CFTR. Kuyafana ne-VX-770 ukuze ikwazi ukufumana ityuwa egeleza ngokufanelekileyo kwiiseli, kodwa isebenza ngokungafaniyo. Ukuba isebenza ngendlela abaphandi abathemba ngayo ukuba, i-VX-809 iya kuvulwa iziteshi ze-chloride ngokuhambisa iprotheni yeCFTR kwindawo yayo efanelekileyo kwi-membrane yeselwandle.
Miglustat
I-Miglustat yiyilisi eyenziwa yi-Actelion Pharmaceuticals esele isetyenziselwa ukunyanga ezinye iimeko, kodwa ngoku iqhutyelwa ukusetyenziswa kubantu abane- cystic fibrosis abaneeikopi ezimbini ze-ΔF508-CFTR. Isifundo sincinci (sinobathathi-nxaxheba aba-15 kuphela), kodwa ngoku iziphumo zithembisile. I-Miglustat iye yakwazi ukuguqula isiphene seCFTR nokubuyisela umsebenzi oqhelekileyo kwiiseli.
Ataluren
I-Ataluren, eyayibizwa ngokuba yi-PTC124, ifundiswa yi-PTC Therapeutics njengokuba kunokwenzeka ukuba unyango lwabantu abaneCF abanokuguqulwa kwamandla. Ngokungatshintshi kweengcamango, iqela le "khowudi" libonakala phakathi kwekhowudi evamile kwi-CFTR gene.
Ikhowudi engenamsebenzi ibonisa uphawu lokuyeka, ukuthintela iiseli ekufundeni nayiphi na ikhowudi eyenzeka emva kwayo. I-Ataluren inokukwazi ukulungisa loo ngxaki ngokunceda iiseli ukuba zingayinaki umqondiso wokuma kwaye ziqhubeke zifunda ikhowudi eyenzeka emva kwayo, ngoko ibuyisela umsebenzi oqhelekileyo kwiiseli.
> Imithombo:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "I-CF yokuphefumula i-Cell Epithelial Cell Uphethwe yi-Miglustat ifumana i-Non-CF njengeFenotype". I-American Journal of Cell Respiratory and Biology Biology . Agasti 2009.
> I-Cystic Fibrosis Foundation. Juni 2009. Iipayipi lokuPhuhlisa iMida. 24 uJulayi 2009.
> Zhang L, Inkomfa B, uGabriel SE, iBurkett S, Yan Y, et al. 2009 "I-CFTR Ukunikezelwa kwi-25% yee-Epithelial Cell cells ibuyisela iiRhafu eziqhelekileyo zokuThuthwa kweMucus kwi-Cystic Fibrosis Airway Epithelium". I-PLoS Biol 7 (7): e1000155. i-doi: 10.1371 / iphephandaba.pbio.1000155. 24 uJulayi 2009.