I-Donilorsen (API)
Isicelo socwaningo:
I-Donilorsen API iyi-antisense oligonucleotide (ASO) ephenywayo yokwelashwa kwe-angioedema yofuzo (HAE) kanye nezimo zokuvuvukala ezihlobene. Kufundwa kumongo wezindlela zokwelapha eziqondiswe ku-RNA, okuhloswe ngazo ukunciphisa ukubonakaliswa kwei-plasma prekallikrein(KLKB1 mRNA). Abacwaningi basebenzisa i-Donidalorsen ukuhlola izindlela zokuthulisa izakhi zofuzo, i-pharmacokinetics encike kumthamo, kanye nokulawulwa kwesikhathi eside kokuvuvukala okuhambisana ne-bradykinin.
Umsebenzi:
I-Donidalorsen isebenza ngokubopha ngokukhetha kuKLKB1i-mRNA, inciphisa ukukhiqizwa kwe-plasma prekallikrein - i-enzyme eyinhloko ohlelweni lwe-kallikrein-kinin ebangela ukuvuvukala nokuvuvukala ku-HAE. Ngokwehlisa amazinga e-kallikrein, i-Donidalorsen isiza ukuvimbela ukuhlaselwa kwe-HAE futhi yehlise umthwalo wezifo. Njenge-API, isebenza njengengxenye yokwelapha eyinhloko ekuthuthukisweni kwemithi esebenza isikhathi eside, elawulwa ngaphansi kwesikhumba ye-HAE.