Givosiran

Givosiran (API)

Aikace-aikacen Bincike:
Givosiran API wani ƙaramin RNA ne mai shiga tsakani (siRNA) wanda aka yi nazari don maganin cututtukan hanta mai tsanani (AHP). Yana musamman hari daALAS1gene (aminolevulinic acid synthase 1), wanda ke da hannu a cikin hanyar heme biosynthesis. Masu bincike suna amfani da Givosiran don yin bincike kan tsoma bakin RNA (RNAi), hanyoyin kwantar da hankali na hanta da ke da niyya, da daidaita hanyoyin hanyoyin rayuwa da ke cikin porphyria da cututtukan ƙwayoyin cuta masu alaƙa.

Aiki:
Ayyukan Givosiran ta hanyar rage yawan maganaALAS1a cikin hepatocytes, don haka yana rage yawan tari na tsaka-tsakin heme mai guba kamar ALA (aminolevulinic acid) da PBG (porphobilinogen). Wannan yana taimakawa hana hare-haren neurovisceral da ke hade da m hepatic porphyria. A matsayin API, Givosiran shine kayan aikin magunguna masu aiki a cikin hanyoyin jiyya na tushen RNAi wanda aka tsara don samar da kulawa na dogon lokaci na AHP tare da gudanarwa na subcutaneous.