Nā API oligonucleotide
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ʻO ka sodium Inclisiran
ʻO Inclisiran sodium API (Active Pharmaceutical Ingredient) ke aʻo nui ʻia ma ke kahua o ka RNA interference (RNAi) a me nā therapeutics cardiovascular. Ma ke ʻano he siRNA pālua ʻelua e kuhikuhi ana i ka ʻano PCSK9, hoʻohana ʻia ia i ka noiʻi preclinical a me ka noiʻi lapaʻau e loiloi i nā hoʻolālā hoʻopaʻa ʻana i nā gene-silencing lōʻihi no ka hoʻohaʻahaʻa ʻana i ka LDL-C (low-density lipoprotein cholesterol). He mea hoʻohālike ia no ka noiʻi ʻana i nā ʻōnaehana lawe siRNA, kūpaʻa, a me nā therapeutics RNA i hoʻopaʻa ʻia i ka ate.
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Donidalorsen
ʻO Donidalorsen API kahi antisense oligonucleotide (ASO) ma lalo o ka hoʻokolokolo ʻana no ka mālama ʻana i ka angioedema hereditary (HAE) a me nā kūlana inflammatory pili. Ua aʻo ʻia i loko o ka pōʻaiapili o nā lāʻau lapaʻau i hoʻopaʻa ʻia i ka RNA, me ka manaʻo e hōʻemi i ka hōʻike ʻana oplasma prekallikrein(KLKB1 mRNA). Hoʻohana ka poʻe noiʻi iā Donidalorsen e ʻimi i nā mīkini silencing gene, pharmacokinetics hilinaʻi dose, a me ka hoʻomalu lōʻihi o ka ʻāʻī ʻana o bradykinin.
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Fitusiran
ʻO Fitusiran API kahi RNA interfering liʻiliʻi liʻiliʻi (siRNA) i noiʻi nui ʻia ma ke kahua o ka hemophilia a me nā maʻi coagulation. Huli ia i kaantithrombin (AT a i ʻole SERPINC1)gene i loko o ke ake e ho'ēmi i ka hana antithrombin. Hoʻohana ka poʻe noiʻi iā Fitusiran e ʻimi i nā hana RNA interference (RNAi), silencing gene kikoʻī kikoʻī o ka ate, a me nā hoʻolālā therapeutic novel no ka hoʻoponopono hou ʻana i ka coagulation i nā maʻi hemophilia A a me B, me ka ʻole a i ʻole nā mea hoʻopaneʻe.
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Givosiran
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.
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Plozasiran
ʻO Plozasiran API kahi RNA interfering liʻiliʻi liʻiliʻi (siRNA) i hoʻomohala ʻia no ka mālama ʻana i ka hypertriglyceridemia a me nā maʻi cardiovascular a me ka metabolic pili. Huli ia i kaAPOC3gene, ka mea e hoʻopili i ka apolipoprotein C-III, kahi mea hoʻoponopono koʻikoʻi o ka metabolism triglyceride. I ka noiʻi ʻana, hoʻohana ʻia ʻo Plozasiran e aʻo i nā hoʻolālā hoʻohaʻahaʻa lipid e pili ana i ka RNAi, ka kikoo-silencing kikoʻī, a me nā lāʻau lōʻihi no nā kūlana e like me ka familial chylomicronemia syndrome (FCS) a me ka dyslipidemia hui ʻia.
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Zilebesiran
ʻO Zilebesiran API kahi noiʻi liʻiliʻi liʻiliʻi interfering RNA (siRNA) i kūkulu ʻia no ka mālama ʻana i ka hypertension. Huli ia i kaAGTgene, e hoʻopili ana i ka angiotensinogen—he mea nui o ka ʻōnaehana renin-angiotensin-aldosterone (RAAS). Ma ka noiʻi, hoʻohana ʻia ʻo Zilebesiran e aʻo i nā ala hoʻoheheʻe gene no ka mālama ʻana i ke koko lōʻihi, nā ʻenehana lawe RNAi, a me ka hana ākea o ke ala RAAS i nā maʻi cardiovascular a me ka renal.