Kulonjeza Zipatala Zatsopano
Pakadali pano, palibe mankhwala a cystic fibrosis (CF), koma ofufuza akugwira ntchito mwakhama kuti apeze imodzi. Asayansi ali pafupi tsopano kuposa kale lonse. Pazaka makumi angapo zapitazo, kufufuza kwakukulu kwachititsa kuti pakhale chithandizo chamankhwala atsopano komanso mankhwala omwe athandiza kwambiri moyo ndi moyo wabwino. Zonsezi zatsegula zitseko za kafukufuku watsopano zomwe zingachititse kuchiza.
Zotsatirazi ndi zina chabe za machiritso omwe angaphunzire.
Gene Therapy
Mu 1989, jini yoyambitsa cystic fibrosis inapezedwa: jini la CFTR . Kupeza uku kunali kosangalatsa kwa gulu la CF. Ambiri ankakhulupirira kuti kupezeka kumeneku kungachititse kuti munthu adziritse matenda okhudza jini.
Mwatsoka, izi sizinachitikebe, koma osati chifukwa chosowa kuyesera. Kafukufuku wambiri wachitidwa pofuna kuyesa ndikukonza chilema cha genetic koma palibe chomwe chinawoneka bwino. Vuto lalikulu kwambiri la matenda a jini pakalipano akupeza vector yomwe ingathe kunyamula geni yolongosoledwa.
Komabe pangakhalebe chiyembekezo cha gene genetic, ngakhale. Mu July 2009, gulu la ofufuza ku yunivesite ya North Carolina ku Chapel Hill linali ndi zotsatira zabwino kwambiri pogwiritsira ntchito kachilombo kozizira kamodzi monga chithunzithunzi chotengera jini m'matumba a mapapu. Gulu lofufuzira tsopano likugwiritsa ntchito njira yochepetsera tizilombo toyambitsa matenda kotero kuti chithandizochi chikhoza kuyesedwa kwa anthu omwe ali ndi cystic fibrosis.
VX-770
VX-770 ndi mankhwala omwe akuyesedwa ndi Vertex Pharmaceuticals kwa anthu omwe ali ndi cystic fibrosis omwe ali ndi kopi imodzi yokha ya kusintha kwa G551D. Mankhwalawa akhoza kwenikweni kulunjika chilema mu jini la CFTR ndikubwezeretsanso njira yotsegulira njira za chloride, motero mchere umatuluka ndi kutuluka mu maselo bwino.
Mosiyana ndi mankhwala a jini, VX-770 sichidzalowetsa jini yolakwika. M'malo mwake, ngati atapambana, VX-770 ikakonza vutoli mu jini yomwe ilipo.
VX-809
VX-809 ndi mankhwala ena omwe akuyesedwa ndi Vertex Pharmaceuticals mwa anthu omwe ali ndi makope awiri a ΔF508-CFTR kusintha. N'chimodzimodzinso ndi VX-770 mmenemo kuti ikhoza kupeza mchere ukuyenda mwa maselo moyenera, koma zimagwira ntchito mosiyana. Ngati ikugwira ntchito momwe ofufuza akuyembekeza kuti izo, VX-809 ikhoza kutsegula chloride njira poyendetsa puloteni ya CFTR kumalo ake oyenera pa memphane yapansi.
Miglustat
Miglustat ndi mankhwala omwe amapangidwa ndi Actelion Pharmaceuticals omwe agwiritsidwa kale ntchito kuti athetse zovuta zina, koma pakalipano akuphunziridwa kuti agwiritsidwe ntchito kwa anthu omwe ali ndi cystic fibrosis omwe ali ndi makope awiri a ΔF508-CFTR kusintha. Phunziroli ndi laling'ono (limakhala ndi anthu 15 okha), koma mpaka pano zotsatira zakhala zikulonjeza. Miglustat yatha kuthetsa vuto la CFTR ndikubwezeretsanso maselo ambiri.
Ataluren
Ataluren, yomwe poyamba idatchedwa PTC124, ikuphunziridwa ndi PTC Therapeutics ngati njira yothetsera anthu omwe ali ndi CF omwe ali ndi kusintha kwachabechabe. Mukusintha kwachabechabe, chidutswa cha "gibberish" code chikupezeka pakati pa chizolowezi chodziwika mu gene ya CFTR.
Ndondomeko yopanda pake imakhala ngati chizindikiro choyimitsa, kuteteza maselo kuti asawerenge chikhombo chilichonse chomwe chimachitika pambuyo pake. Ataluren akhoza kuthetsa vutoli pothandiza maselo kunyalanyaza chizindikiro choyimira ndikupitiriza kuwerenga mndandanda womwe umapezeka pambuyo pake, motero kubwezeretsa ntchito yachibadwa kwa maselo.
> Zotsatira:
> C. Norez, F. Antigny, S. Noel, C. Vandebrouck, F. Becq. "CF Respiratory Epithelial Cell Chronicle Kutengedwa ndi Miglustat Akupeza Non-CF Monga Phenotype". Journal ya American Respiratory Cell ndi Biology Molecular . August 2009.
> Cystic Fibrosis Foundation. June 2009. Pipeline yopititsa patsogolo mankhwala. 24 July 2009.
> Zhang L, Batani B, Gabriel SE, Burkett S, Yan Y, et al. 2009 "CFTR Kuperekedwa kwa 25% ya Ma cell Epithelial Wowonjezera Kubwezeretsa Zowonongeka za Mucus Transport ku Human Cystic Fibrosis Airway Epithelium". Chipangizo cha PLoS 7 (7): e1000155. lembani: 10.1371 / magazini.pbio.1000155. 24 July 2009.