ʻO ka Frontier Nui o nā molekole liʻiliʻi--RNA hulina

2024-03-07 15:49:45

Ua manaʻo lōʻihi ʻo ka hoʻohana ʻana i nā kemika e hahai i ka RNA he mea lapuwale ma muli o kāna mau waiwai. Ke holomua ka poʻe ʻepekema, ʻo ka mea maʻamau ma ka pōʻino.

I nā makahiki he ʻumi i hala iho nei, no ka laʻana, ua ʻike ʻo Merck i kahi lāʻau antibiotic hoʻokolohua i ka hoʻomohala ʻana e poloka ana i kahi ʻano RNA bacteria. Ua hina ʻo Pfizer hiki i kāna lāʻau lapaʻau ke hoʻopilikia i ka unuhi ʻana o kahi protein e hoʻoponopono ai i ka cholesterol.

I kēia manawa, manaʻo nā hui lāʻau e hana i kēlā. Me ke kōkua o nā ʻenehana hoʻonohonoho ʻoi aku ka maikaʻi, nā ʻano kānana, a me ka ʻike ākea o ka RNA, hiki i nā mea noiʻi ke hopu maʻalahi i ke ʻano o nā molekele ʻike a hoʻolālā i nā lāʻau lapaʻau e pili ana iā lākou. Inā kūleʻa, hiki iā lākou ke kōkua i ka hoʻokō ʻana i nā pahuhopu maʻi ʻaʻole hiki i nā molekala liʻiliʻi ke hiki i kēia manawa, e hāʻawi ana i nā ala hou e mālama ai i nā maʻi neurodegenerative, ka maʻi kanesa a me nā kūlana ʻē aʻe. 18383aa279538923fe77cddddlkv

01 He aha nā molekole liʻiliʻi e kuhikuhi ana i ka RNA?

ʻO nā molekole liʻiliʻi he mau lāʻau kemika i hoʻopaʻa pinepine ʻia i nā protein, pale a hoʻololi paha i ke ʻano o kā lākou hana. Ma muli o ko lākou nui, hiki i nā molekala liʻiliʻi ke hiki i ka hapa nui o nā ʻiʻo i loko o ke kino o ke kanaka, paheʻe i loko o nā cell, a hoʻopaʻa i ka concave a me ka convex site o ka pahu hopu.

ʻO lākou ka pōhaku kihi o ka ʻoihana lāʻau lapaʻau a lilo i ka hapa nui o nā lāʻau lapaʻau ma ka mākeke. Akā ua kaupalena ʻia ka laulā o nā molekala liʻiliʻi. Ma kahi kokoke i 20,000 mau genes i ʻike ʻia, ma kahi o 3,000 i manaʻo ʻia he mea hoʻopau ʻia, ʻo ia hoʻi hiki ke hoʻopaʻa ʻia me nā lāʻau lapaʻau. ʻO nā protein a lākou e hana ai, he mau haneli wale nō ka mea i manaʻo ʻia no nā lāʻau lapaʻau. ʻAʻole loaʻa nā "ʻeke" ʻē aʻe he nui i nā molekala liʻiliʻi e hoʻokomo ai, e ʻoi aku ka paʻakikī o ka loaʻa ʻana.

ʻO ka huli ʻana i nā molekole RNA e kōkua i ka hana ʻana i nā protein pili i ka maʻi hiki ke hāʻawi i nā mea hana lāʻau i kahi ala ʻē aʻe. Ma ke kumumanaʻo, hiki i ka lāʻau lapaʻau ke pale i ka hana ʻana o kahi protein hiki ke hoʻopōʻino a hana paha i nā protein ʻoi aku ka maikaʻi.

02 Li'ili'i mole mole pono ʻokoʻa o ka huli ʻana i ka RNA.

ʻO nā lāʻau lapaʻau e hoʻopilikia i ka RNA ua hōʻike ʻia he mau mea hana pono no ka mālama ʻana i nā ʻano maʻi. ʻO kekahi mai ka Alnylam Pharmaceuticals, nāna i hoʻokumu i kahi ʻano i kapa ʻia ʻo RNA interference e hoʻohana ana i nā molekele RNA synthetic liʻiliʻi e hoʻopaʻa i nā genes, e pale ana iā lākou mai ka hana ʻana i nā protein ʻino.

ʻO kekahi ala like, hoʻohana ʻia e Ionis Pharmaceuticals a me nā hui ʻē aʻe, hoʻohana i nā ʻāpana o ka nucleic acid i kapa ʻia ʻo antisense oligonucleotides e hoʻoponopono a hoʻopau i ka hana ʻana o nā protein. Ua holomua nā hui ʻelua i ka noiʻi RNA, ʻoi aku ka huli ʻana i nā lāʻau lapaʻau no ka mālama ʻana i nā maʻi ʻano like ʻole e like me ka spinal muscular atrophy a me ka thyroid transamyloidosis, a me nā kūlana maʻamau e like me ka cholesterol kiʻekiʻe. Hiki i nā hopena o ka lāʻau ke hoʻomau no nā mahina i ka manawa.

ʻOiai ua loaʻa ka holomua nui i ka hāʻawi ʻana i kēia mau lāʻau lapaʻau RNA i loko o ke kino, ua kaupalena ʻia lākou i nā pahuhopu maʻi i loko o ke ake, ʻo ia hoʻi ka nui o nā maʻi hiki ʻole ke mālama ʻia.

03 ʻO nā molekala liʻiliʻi e huli ana i ka RNA ka pae aʻe.

ʻOiai ua komo ka nui o nā lāʻau lapaʻau i kūkulu ʻia ma nā ʻenehana RNA ʻē aʻe i ka mākeke i nā makahiki i hala iho nei, ʻo nā molekole liʻiliʻi e hiki ke hoʻokō i ka RNA ke hoʻomaka wale nei e hōʻike i ko lākou hiki.

ʻO ka mea mua i hana pēlā ʻo Evrysdi, kahi lāʻau i hoʻomohala ʻia e Roche a me PTC Therapeutics a ʻae ʻia i 2020 e kōkua i ka hana ʻana i kahi protein i nele i nā mea maʻi me ka spinal muscular atrophy. ʻO kahi lāʻau maʻi maʻi Huntington hoʻokolohua mai Novartis i kapa ʻia ʻo branaplam aia pū kekahi i nā hoʻokolohua kanaka, akā hiki i nā pilikia palekana hou ke keʻakeʻa i kona holomua.

I kēia manawa, ʻaʻohe mea hoʻomaka e hoʻomohala ana i nā molekole RNA-targeting liʻiliʻi i lawe i kahi lāʻau lapaʻau i nā hoʻokolohua lapaʻau. Akā ke hoʻomaka nei nā paipu o kekahi ʻoihana. ʻIke ʻia ʻo Skyhawk i ka pō o ke komo ʻana i nā hoʻokolohua lapaʻau, a ua ʻōlelo mua ka ʻoihana e hoʻolālā ana e hoʻomaka i ka neʻe ʻana i kāna lāʻau lapaʻau mua i nā hoʻokolohua kanaka i kekahi manawa o kēia makahiki.