Ho tšepisa Phekolo e Ncha
Ho fihlela joale, ha ho na pheko ea cystic fibrosis (CF), empa bafuputsi ba sebetsa ka thata ho e fumana. Bo-rasaense ba haufi haholo ho feta leha e le neng pele. Lilemong tse mashome a mabeli a ho qetela, lipatlisiso tse ngata li entse hore ho hlahisoe lithethefatsi le meriana e mengata e mecha e ntlafetseng haholo bophelo bo lekaneng le boleng ba bophelo. Tsena tsohle li butse menyetla ea lipatlisiso tse ncha tse ka lebisang phekolong.
Lintlha tse latelang ke tse ling tsa phekolo e ka fumanoang.
Gene Therapy
Ka 1989, ho ile ha fumanoa liphatsa tsa lefutso tse bakang cystic fibrosis: liphatsa tsa lefutso la CFTR . Ho sibolloa hona ho ne ho thabisa sechaba sa CF. Ba bangata ba ne ba lumela hore ho sibolloa ho tla lebisa phekolong ka ho sebelisa phekolo ea lefutso.
Ka bomalimabe, ha ho e-s'o etsahalloe, empa ha ho joalo ka ho hloka teko. Ho entsoe lithuto tse ngata ho leka le ho lokisa bothata ba liphatsa tsa lefutso empa ha ho le e 'ngoe ea tsona e atlehileng. Bothata bo boholo ka ho phekola liphatsa tsa lefutso ho fihlela hona joale bo fumane vector e ka sebetsang ka katleho liphatsa tsa lefutso ho lisele.
Ho ntse ho ka 'na ha e-ba le tšepo ea phekolo ea lefutso, leha ho le joalo. Ka July 2009, sehlopha sa bafuputsi Univesithing ea North Carolina e Chapel Hill se bile le liphello tse ntle haholo ho sebelisa kokoana-hloko e tloaelehileng ea serame e le sekheo sa ho fetisetsa liphatsa tsa lefutso lihlahisoa tsa laboratori tsa lisele tsa matšoafo. Sehlopha sa lipatlisiso se se se sebetsana le mokhoa oa ho fokolisa kokoana-hloko e batang e le hore kalafo e ka lekoa ho batho ba nang le cystic fibrosis.
VX-770
VX-770 ke lithethefatsi tse nkiloeng ke Vertex Pharmaceuticals ho batho ba nang le cystic fibrosis ba nang le bonyane kopi e le 'ngoe ea phetoho ea G551D. Ka sebele sethethefatsi sena se ka khona ho lebisa phoso ea lefutso la liphatsa tsa lefutso la CFTR le ho khutlisetsa matla a eona a ho bula liseishene tsa chloride, kahoo ho lumella letsoai ho phalla le ho tsoa ka lisele hantle.
Ho fapana le phekolo ea liphatsa tsa lefutso, VX-770 e ke ke ea nkela sebaka sa lefutso le nang le bokooa. Ho e-na le hoo, haeba e atlehile, VX-770 e tla lokisa bothata boteng ba lefutso.
VX-809
VX-809 ke lithethefatsi tse ling tse nkiloeng ke Vertex Pharmaceuticals ho batho ba nang le likopi tse peli tsa phetoho ea ΔF508-CFTR. E tšoana le VX-770 ka hore e ka khona ho fumana letsoai le phallelang ka lisele ka mokhoa o nepahetseng, empa le sebetsa ka tsela e sa tšoaneng. Haeba e sebetsa ka tsela eo bafuputsi ba nang le tšepo ea hore e tla etsahala, VX-809 e ne e tla bula litsela tsa chloride ka ho tsamaisa protheine ea CFTR sebakeng sa eona se nepahetseng litebeleng tsa moea.
Miglustat
Miglustat ke lithethefatsi tse entsoeng ke Actelion Pharmaceuticals tse seng li sebelisoa ho phekola maemo a mang, empa hona joale li ntse li ithutoa bakeng sa ho sebelisoa ho batho ba nang le cystic fibrosis ba nang le likopi tse peli tsa phetoho ea ΔF508-CFTR. Thuto e nyane (e na le litho tse 15 feela), empa ho fihlela joale liphello li tšepisitse. Miglustat e khonne ho fetola sekoli sa CFTR le ho tsosolosa mosebetsi o tloaelehileng ho lisele.
Ataluren
Ataluren, eo pele e neng e bitsoa PTC124, e ntse e ithutoa ke PTC Therapeutics e le pheko ea batho ba nang le CF ba nang le liphetoho tse se nang thuso. Ka liphetoho tse se nang moelelo, karoloana ea "code" e hlaha ka har'a code e tloaelehileng ka lekhetlo la CFTR.
Khoutu e se nang boitsoaro e sebetsa joaloka letšoao le emang, e thibela lisele hore li se ke tsa bala khoutu leha e le efe e hlahang ka mor'a eona. Ataluren e ka khona ho lokisa bothata boo ka ho thusa lisele hore li hlokomolohe letšoao le emang le ho tsoela pele ho bala khoutu e hlahang ka mor'a eona, kahoo e tsosolosa mosebetsi o tloaelehileng liseleng.
> Mehloli:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "CF Epithlial Cell Ka Kakaretso e Tšoaroang ke Miglustat e Fumana e Ntle ho CF joaloka Phenotype". Journal of American Journal of Cell Respiratory and Biology Molecular . August 2009.
> Motheo oa Cystic Fibrosis. June 2009. Pipeline ea Ntšetso-pele ea Lithethefatsi. 24 Phupu 2009.
> Zhang L, Button B, Gabriel SE, Burkett S, Yan Y, et al. 2009 "CFTR e fetiselitsoe ho 25% ea li-cellitheshene tsa" Epithelial Surface "e tsosolosa litefiso tse tloaelehileng tsa Mucus Transport ho Human Cystic Fibrosis Airway Epithelium". PLoS Biol 7 (7): e1000155. doi: 10.1371 / koranta.pbio.1000155. 24 Phupu 2009.