Achivimbisa Zvitsva Zvitsva
Kusvika ikozvino, hapana mushonga we cystic fibrosis (CF), asi vatsvakurudzi vari kushanda nesimba kuti vawane imwe. Masayendisiti ari pedyo ikozvino kupfuura kare. Pamusoro pemakore makumi maviri apfuura, boka guru rekutsvakurudza rakatungamirira kukurudzirwa kwemishonga mitsva nemishonga yakanyatsogadzirisa hupenyu hwehupenyu hwose uye hupenyu hwehupenyu. Zvose izvi zvakazarura mikova yekutsvakurudza kutsva kunogona kutungamirira kune chirwere.
Zvinotevera ndezvimwe zvezvirwere zvinogona kuwanikwa.
Gene Therapy
Muna 1989, jena rinokonzera kukonzera cystic fibrosis yakawanikwa: CFTR gene . Uku kuwanikwa kwaifadza vanhu veCF. Vakawanda vaidavira kuti kuwanikwa kwaizotungamirira kune kurapwa kuburikidza nejena therapy.
Zvinosuruvarisa, izvo hazvina kumboitika, asi kwete nekuda kwekuedza. Zvidzidzo zvakasiyana-siyana zvakaitwa kuedza nekugadzirisa kukanganisa kweiyo genetic asi hapana chimwe chazvo chakabudirira. Dambudziko guru rejene therapy kusvika ikozvino rave richiwana vector iyo inokwanisa kutakura zvakakwana geni rakagadzirirwa kumasero.
Panogona kunge kune tariro ye gene gene, kunyange zvakadaro. Muna July 2009, boka revanotsvakurudza paYunivhesiti yeNorth Carolina kuChapel Hill raiva nemigumisiro yakanaka kwazvo kushandisa chirwere chinotonhora chinotonhorera sevector kuendesa jeni mu laboratory samples yemapapu. Dare rekutsvakurudza ikozvino rinoshanda munzira yekuderedza firusi inotonhora kuitira kuti kurapwa kunogona kuongororwa kune vanhu vane cystic fibrosis.
VX-770
VX-770 imishonga iri kuongororwa neVertex Pharmaceuticals kune vanhu vane cystic fibrosis vane ine kana imwe kopi imwe yeG551D kuchinja. Iyo mishonga inogona kunge yakakwanisa kutarisa kukanganisa muCFTR gene uye inodzorera kukwanisa kwayo kuzarura michina yekloride, nokudaro zvichiita kuti munyu upinde mukati uye kunze kwemasero zvakanaka.
Kusiyana neine gene genetic, VX-770 haizogadziri jena rakashata. Pane kudaro, kana akabudirira, VX-770 yaizogadzirisa dambudziko iri mune jena iripo.
VX-809
VX-809 imwe mishonga iri kuongororwa neVertex Pharmaceuticals kuvanhu vane makopi maviri eAF508-CFTR mutation. Izvo zvakafanana neVX-770 mukuti inogona kukwanisa kuisa munyu ichiyerera mumasero zvakakodzera, asi inoshanda zvishomanana zvakasiyana. Kana ikashanda nenzira iyo vatsvakurudzi vanotarisira kuti ichaita, VX-809 yaizozarura mishonga yekloride nekufambisa protein yeCFTR panzvimbo yayo yakakodzera panzira yemhepo membrane.
Miglustat
Miglustat imishonga inogadzirwa neActelion Pharmaceuticals iyo inenge ishandiswa kurapa mamwe mamiriro ezvinhu, asi ikozvino iri kuongororwa kushandiswa kune vanhu vane cystic fibrosis vane mabhuku maviri eAF508-CFTR mutation. Chidzidzo chacho chiduku (chinoumbwa nevatori vega 15 chete), asi kusvika zvino zvave zvichivimbisa. Miglustat yakwanisa kugadzirisa chinokonzera CFTR uye kudzorera basa rakakwana kumasero.
Ataluren
Ataluren, iyo yaimbozivikanwa sePTC124, iri kuongororwa nePTC Therapeutics sechinhu chinogona kurapwa kune vanhu vane CF avo vasina maturo. Mukunyengedza kusina maturo, chidimbu che "gibberish" code inowanikwa mukati mekodhi yekodhi muCFTR gene.
Nhamba yekusina maturo inoshanda sechiratidzo chekumira, kudzivirira masero kuti arege kuverenga chero chipi zvacho chinowanikwa mushure maro. Ataluren inogona kukwanisa kugadzirisa chinetso ichocho nokubatsira masero kuti asatarise chiratidzo chekumira uye kuramba achiverenga kero inoitika mushure mayo, nokudaro kudzorera basa rakanaka kumasero.
> Sources:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "CF Kufuridzirwa Epithelial Cell Isingaperi Inotariswa neMiglustat Inowana I-CF Sezvinonzi Phenotype". American Journal of Respiratory Cell uye Molecular Biology . August 2009.
> Cystic Fibrosis Foundation. June 2009. Drug Development Pipeline. 24 July 2009.
> Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, et al. 2009 "CFTR Kuendeswa kune 25% yeEpithelial Cell Cells Inovandudza Nhamba Dzakakwana dzeMucus Transport kusvika kuCys Cystic Fibrosis Airway Epithelium". PLoS Biol 7 (7): e1000155. doi: 10.1371 / journal.pbio.1000155. 24 July 2009.