Givosiran

Givosiran (API)

Noi noiʻi:
ʻO Givosiran API kahi RNA interfering liʻiliʻi liʻiliʻi (siRNA) i aʻo ʻia no ka mālama ʻana i ka porphyria hepatic acute (AHP). Kuhi nui ia i kaALAS1gene (aminolevulinic acid synthase 1), i komo i ke ala biosynthesis heme. Hoʻohana nā mea noiʻi iā Givosiran e noiʻi i nā lāʻau lapaʻau e pili ana i ka RNA interference (RNAi), silencing gene i hoʻopaʻa ʻia i ka ate, a me ka modulation o nā ala metabolic e pili ana i ka porphyria a me nā maʻi genetic pili.

Hana:
Hana ʻo Givosiran ma ka hōʻemi ʻana i ka ʻōlelo oALAS1i loko o nā hepatocytes, ma laila e hoʻemi ai i ka hōʻiliʻili ʻana o nā mea ʻawaʻawa heme e like me ALA (aminolevulinic acid) a me PBG (porphobilinogen). Kōkua kēia i ka pale ʻana i nā hōʻeha neurovisceral e pili ana me ka porphyria hepatic acute. Ma ke ʻano he API, ʻo Givosiran ka mea lāʻau lapaʻau ikaika i ka RNAi-based therapeutics i hoʻolālā ʻia e hāʻawi i ka mana lōʻihi o AHP me ka hoʻokele subcutaneous.