Ukuthembisa Ukwelapha Okusha
Kuze kube manje, ayikho ikhambi ye- cystic fibrosis (CF), kodwa abacwaningi basebenza kanzima ukuthola eyodwa. Ososayensi basondele manje kunanini ngaphambili. Emashumini eminyaka edlule, ucwaningo oluningi luye lwaholela ekuthuthukiseni izidakamizwa eziningi nezinqubo ezintsha zokwelapha ezithuthukise ngokubili isikhathi sokuphila kanye nemfanelo yokuphila. Konke lokhu kuvulwe iminyango yocwaningo olusha olungabangela ukwelashwa.
Lokhu okulandelayo kungenye yezokwelapha okukhona okufundiswayo.
I-Gene Therapy
Ngo-1989, isakhi sofuzo esibangela ukuba i-cystic fibrosis sitholwe: isakhi se-CFTR . Lokhu kutholakala kwaba mnandi kumphakathi we-CF. Abaningi bakholelwa ukuthi ukutholakala kuzoholela ekwelapheni ngokusebenzisa i-gene therapy.
Ngeshwa, lokho akukenzekanga okwamanje, kodwa hhayi ngenxa yokuntuleka kokuzama. Kuye kwenziwa izifundo eziningi ukuzama ukulungisa ukukhubazeka kwezakhi zofuzo kodwa akekho oye waphumelela. Inkinga enkulu yokwelashwa kwegciwane okwamanje iye yathola i-vector engakwazi ukuthwala igalelo elungisiwe kumaseli.
Kungase kube namanje ithemba nge-gene therapy, noma kunjalo. NgoJulayi 2009, iqembu labacwaningi eYunivesithi yaseNorth Carolina e-Chapel Hill lalinemiphumela emihle kakhulu ngokusebenzisa igciwane elibandayo elivuthayo njenge-vector ukudlulisa igalelo kuma-laboratory amasampula amaphaphu. Ithimba lokucwaninga selisebenza ngendlela yokunciphisa igciwane elibandayo ukuze ukwelashwa kuhlolwe kubantu abane-cystic fibrosis.
I-VX-770
I-VX-770 yisidakamizwa esivivinywa yi-Vertex Pharmaceuticals kubantu abane-cystic fibrosis abane okungenani ikhophi eyodwa yokuguqulwa kwe-G551D. Isidakamizwa kungenzeka empeleni sikwazi ukukhomba isici kulesisindo se-CFTR futhi siphinde sikwazi ukuvula iziteshi ze-chloride, ngaleyo ndlela sivumele usawoti uphelele kahle futhi uphume kwamaseli.
Ngokungafani ne-gene therapy, i-VX-770 ngeke isuse isakhi sofuzo. Kunalokho, uma uphumelela, i-VX-770 izoyilungisa inkinga kuleso sakhi sekhona.
I-VX-809
I-VX-809 ingenye imithi ehlolwa yi-Vertex Pharmaceuticals kubantu abanamakhophi amabili we-ΔF508-CFTR ishintsho. Kufana ne-VX-770 ngoba kungenzeka ukwazi ukuthola usawoti ogeleza ngokuqondile ngamaseli, kodwa usebenza kancane kancane. Uma isebenza ngendlela abacwaningi abayethemba ngayo ukuthi, i-VX-809 izovula iziteshi ze-chloride ngokuhambisa iprotheyini ye-CFTR endaweni yayo efanele emgqeni womoya.
Miglustat
I-Miglustat yiyidakamizwa eyenziwa yi-Actelion Pharmaceuticals esevele isetshenziswa ukuphatha ezinye izimo, kodwa okwamanje ihlolwe ukusetshenziswa kubantu abane- cystic fibrosis abanamakhophi amabili e-ΔF508-CFTR. Ucwaningo luncane (lubandakanya abahlanganyeli abangu-15 kuphela), kodwa kuze kube manje imiphumela isethembisile. I-Miglustat iye yakwazi ukuguqula isiphambeko se-CFTR futhi ubuyisele umsebenzi ojwayelekile kumaseli.
Ataluren
I-Ataluren, eyayibizwa ngokuthi i-PTC124, ifundiswa yi-PTC Therapeutics njengendlela yokwelashwa okungenzeka kubantu abane- CF abanezinkinga ezingenangqondo. Ngezinguquko ezingenangqondo, ucezu lwekhodi "ye-gibberish" livela phakathi kwekhodi evamile kulesisindo se-CFTR.
Ikhodi engenamuntu iyasebenza njengesibonakaliso sokuma, ivimbela amangqamuzana ukuthi afunde noma iyiphi ikhodi eyenzeka ngemva kwayo. I-Ataluren ingakwazi ukulungisa leyo nkinga ngokusiza amangqamuzana ukuba angayinaki uphawu lokuma futhi aqhubeke efunda ikhodi eyenzeka emva kwayo, ngaleyo ndlela abuyisele umsebenzi ojwayelekile kumaseli.
> Imithombo:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "I-CF Epithlial Cell Cell Chronically Yathathwa Miglustat Yathola A Non-CF Like Phhenotype". I-American Journal of Cell Respiratory and Biology Molecular . Agasti 2009.
> I-Cystic Fibrosis Foundation. Juni 2009. Iphayiphu yokuthuthukiswa kwezidakamizwa. 24 Julayi 2009.
> Zhang L, Inkinobho B, Gabriel SE, Burkett S, Yan Y, et al. 2009 "I-CFTR Ukuletha ama-25% ama-Epithelial Cell cells abuyisa amazinga ajwayelekile kaMucus Transport kuya ku-Human Cystic Fibrosis Airway Epithelium". I-PLoS Biol 7 (7): e1000155. doi: 10.1371 / iphephandaba.pbio.1000155. 24 Julayi 2009.