Moeli o moholo oa limolek'hule tse nyane - RNA e shebileng

2024-03-07 15:49:45

Ke khale ho nahanoa hore ho sebelisa lik'hemik'hale ho latela RNA e ne e le lefeela ka lebaka la matla a eona a ho fetoha. Ha bo-rasaense ba atleha, hangata e ba ka tsietsi.

Ka mohlala, lilemong tse leshome tse fetileng, Merck e sibollotse lithibela-mafu tsa liteko tse ntseng li etsoa tse thibelang mofuta oa baktheria ea RNA. Pfizer e ile ea khoptjoa hore moriana oa eona o ka ama phetolelo ea protheine e laolang cholesterol.

Hona joale, lik'hamphani tsa lithethefatsi li ikemiselitse ho etsa joalo. Ka thuso ea mekhoa e ntlafetseng ea tatellano, mekhoa ea ho hlahloba, le kutloisiso e pharaletseng ea RNA, bafuputsi ba ka khona ho tšoara ponahalo ea limolek'hule tsa tlhahisoleseding le ho etsa lithethefatsi tse khomarelang ho tsona. Haeba li atlehile, li ka thusa ho finyella lipakane tsa mafu tseo limolek'hule tse nyenyane li ke keng tsa li finyella hona joale, ho fana ka litsela tse ncha tsa ho phekola mafu a neurodegenerative, kankere le maemo a mang. 18383a279538923fe77cddddlkv

01 Ke limolek'hule life tse nyane tse shebileng RNA?

Limolek'hule tse nyenyane ke lithethefatsi tse thehiloeng lik'hemik'hale tseo hangata li lebisang liprotheine, li thibela kapa li fetola tsela eo li sebetsang ka eona. Ka lebaka la boholo ba tsona, limolek'hule tse nyane li ka fihla liseleng tse ngata tsa 'mele oa motho, tsa thellela liseleng, 'me tsa tlama sebakeng sa concave le convex moo sepheo sa sona.

Ke tsona tse ka sehloohong tsa indasteri ea meriana 'me li etsa boholo ba lithethefatsi tse rekisoang. Empa boholo ba limolek'hule tse nyenyane bo lekanyelitsoe. Ho liphatsa tsa lefutso tse ka bang 20 000 tse tsejoang, tse ka bang 3 000 li nkoa li ka sebelisoa, ho bolelang hore li ka hlaseloa ka lithethefatsi. Har'a liprotheine tseo li li hlahisang, ke tse makholo a seng makae feela tse hlasetsoeng ke lithethefatsi tse seng li ntse li le teng. Liprotheine tse ling tse ngata ha li na "lipokotho" moo limolek'hule tse nyenyane li ka kenang, ho etsa hore ho be thata ho fihla ho tsona.

Ho lebisa limolek'hule tsa RNA tse thusang ho etsa liprotheine tse amanang le mafu ho ka fa baetsi ba lithethefatsi tsela e 'ngoe. Ka khopolo, moriana o ka thibela tlhahiso ea protheine e ka bang kotsi kapa oa hlahisa liprotheine tse molemo haholoanyane.

02 Nyenyane molek'hule molemo o fapaneng oa ho shebisisa RNA.

Lithethefatsi tse sitisang RNA li ipakile e le lithulusi tse sebetsang bakeng sa ho alafa mefuta e mengata ea mafu. E 'ngoe e tsoa ho Alnylam Pharmaceuticals, e qalileng mokhoa o bitsoang tšitiso ea RNA e sebelisang limolek'hule tse nyane tsa maiketsetso tsa RNA ho khutsisa liphatsa tsa lefutso, ho li thibela ho hlahisa liprotheine tse kotsi.

Tsela e 'ngoe e tšoanang, e sebelisoang ke Ionis Pharmaceuticals le lik'hamphani tse ling, e sebelisa lihlopha tsa nucleic acid tse bitsoang antisense oligonucleotides ho laola kapa ho tima tlhahiso ea liprotheine. Lik'hamphani ka bobeli li entse lipatlisiso tsa RNA tse tsoetseng pele, haholo-holo tse lebisang lithethefatsi ho phekola mafu a sa tloaelehang a lefutso a kang spinal muscular atrophy le thyroid transamyloidosis, hammoho le maemo a tloaelehileng a kang k'holeseterole e phahameng. Liphello tsa lithethefatsi li ka nka likhoeli ka nako.

Le hoja ho se ho entsoe tsoelo-pele e khōlō ea ho fana ka litlhare tsena tsa RNA ’meleng, li sa ntse li lekanyelitsoe haholo ho liphofu tsa mafu sebeteng, ho bolelang hore ho ntse ho e-na le mafu a mangata a ke keng a phekoloa.

03 Limolek'hule tse nyane tse shebileng RNA ke mohato o latelang.

Le ha meriana e mengata e hahiloeng holim'a mahlale a mang a RNA e kene 'marakeng lilemong tsa morao tjena, limolek'hule tse nyane tse khonang ho shebana le RNA li qala ho bonts'a bokhoni ba tsona.

Ea pele ea ho etsa joalo ke Evrysdi, sethethefatsi se entsoeng ka kopanelo ke Roche le PTC Therapeutics mme se amohetsoe ka 2020 ho thusa ho hlahisa protheine e haellang ho bakuli ba nang le spinal muscular atrophy. Sethethefatsi sa liteko sa lefu la Huntington se tsoang Novartis se bitsoang branaplam le sona se litekong tsa batho, empa litaba tsa morao-rao tsa polokeho li ka sitisa tsoelo-pele ea eona.

Ho fihlela hajoale, ha ho qalo ea ho hlahisa limolek'hule tse nyane tse shebileng RNA tse nkileng moriana litekong tsa bongaka. Empa liphaephe tsa lik'hamphani tse ling li qala ho sebetsa. Skyhawk e bonahala e le bosiung ba ho kena litekong tsa tleliniki, mme k'hamphani e kile ea re e rera ho qala ho tsamaisa moriana oa eona oa pele litekong tsa batho ka nako e 'ngoe selemong sena.