I-Donilorsen (API)
Isicelo soPhando:
I-Donidalorsen API yi-oligonucleotide ye-antisense (ASO) ephantsi kophando lonyango lwe-angioedema ye-hereditary (HAE) kunye neemeko ezinxulumene nokuvuvukala. Kufundwa kumxholo wonyango olujoliswe kwi-RNA, ejolise ekunciphiseni ukubonakaliswa kweplasma prekallikrein(KLKB1 mRNA). Abaphandi basebenzisa i-Donidalorsen ukuphonononga iindlela zokuthulisa imfuza, i-pharmacokinetics exhomekeke kwidosi, kunye nolawulo lwexesha elide lokudumba kwe-bradykinin-mediated.
Umsebenzi:
Imisebenzi yeDonidalorsen ngokuzibophelela ngokukhethaKLKB1I-mRNA, ukunciphisa ukuveliswa kwe-plasma prekallikrein - i-enzyme ephambili kwinkqubo ye-kallikrein-kinin ejongene nokubangela ukuvuvukala nokuvuvukala kwi-HAE. Ngokunciphisa amanqanaba e-kallikrein, i-Donidalorsen inceda ukukhusela ukuhlaselwa kwe-HAE kunye nokunciphisa umthwalo wesifo. Njenge-API, isebenza njengeyona nto iphambili yonyango ekuphuhliseni ixesha elide, unyango olulawulwa ngaphantsi kwe-HAE.