ʻO nā oligonucleotides nā polymers nucleic acid me nā kaʻina hana kūikawā, me nā antisense oligonucleotides (ASOs), siRNAs (nā RNA liʻiliʻi liʻiliʻi), microRNAs, a me nā aptamers. Hiki ke hoʻohana ʻia nā Oligonucleotides e hoʻololi i ka hōʻike gene ma o nā ʻano hana like ʻole, me ka RNAi, ka hoʻohaʻahaʻa ʻia ma o ka cleavage RNase H-mediated, splicing regulation, noncoding RNA repression, gene activation, a programmed gene editing.
ʻO ka hapa nui o nā oligonucleotides (ASOs, siRNA, a me microRNA) e hoʻohui i ka gen mRNA a i ʻole pre-mRNA ma o ka hoʻohui ʻana i ke kumu hoʻohui, a ma ke ʻano maʻamau hiki ke hoʻololi i ka ʻōlelo o kēlā me kēia gene a me ka protein, me ka nui o nā "non-therapeutic" i pahuhopu. Loaʻa i nā Aptamers ka pilina kiʻekiʻe no ka protein target, e like me ke ʻano tertiary o nā antibodies, ʻaʻole ke kaʻina. Hāʻawi pū ʻo Oligonucleotides i nā pono ʻē aʻe, me ka hana maʻalahi a me nā ʻenehana hoʻomākaukau, nā pōkole hoʻomohala pōkole, a me nā hopena lōʻihi.
Ke hoʻohālikelike ʻia me nā mea hoʻopaneʻe mole liʻiliʻi liʻiliʻi, ʻo ka hoʻohana ʻana i nā oligonucleotides e like me nā lāʻau lapaʻau he ala hou loa. ʻO ka hiki o nā oligonucleotides i nā genetics pololei i hoʻonui i ka hoihoi no nā noi therapeutic i ka maʻi kanesa, ka maʻi cardiovascular, a me nā maʻi maʻamau. ʻO ka ʻae ʻana o FDA hou no Givosiran, Lumasiran a me Viltolarsen e lawe mai iā RNAi, a i ʻole RNA-based therapy, i loko o ke kumu nui o ka hoʻomohala lāʻau.
Ka manawa hoʻouna: Iulai-19-2022