Givosiran

Givosiran (API)

Gusaba Ubushakashatsi:
Givosiran API ni sintetike ntoya yivanga RNA (siRNA) yize kugirango ivure poropiya ikaze ya hepatike (AHP). Irasa cyane cyane iALAS1gene (aminolevulinic acide synthase 1), igira uruhare munzira ya heme biosynthesis. Abashakashatsi bifashisha Givosiran kugira ngo bakore iperereza ku buvuzi bwa RNA (RNAi) bushingiye ku buvuzi, gucecekesha gene igamije umwijima, no guhindura inzira za metabolike zigira uruhare muri porphiriya n'indwara zishingiye ku ngirabuzima fatizo.

Imikorere:
Imikorere ya Givosiran mukugabanya imvugo yaALAS1muri hepatocytes, bityo bikagabanya kwirundanya kwabunzi ba heme nka ALA (aside aminolevulinic) na PBG (porphobilinogen). Ibi bifasha kwirinda ibitero bya neurovisceral bijyana na porphiria ikaze. Nka API, Givosiran nigice gikora imiti muri RNAi ivura igamije gutanga igihe kirekire kugenzura AHP hamwe nubuyobozi bwubutaka.