Nucleic acid tshuaj yog ib hom tshuaj lom neeg tshiab uas siv nucleic acids ua cov khoom xyaw nquag. Lawv tuaj yeem tswj hwm qhov kev qhia lossis kev ua haujlwm ntawm cov hom phiaj ntawm qib molecular, thiab ua tiav cov txiaj ntsig kho mob los ntawm kev kho lossis them nyiaj rau qhov txawv txav ntawm cov noob lossis lawv cov khoom. Cov tshuaj Nucleic acid tau nyiam ntau xyoo tsis ntev los no vim tias lawv muaj peev xwm kho tau ntau yam kab mob, tshwj xeeb tshaj yog cov kab mob tsis tshua muaj tshwm sim los ntawm caj ces tsis xws luag. Hauv tsab xov xwm no, kuv yuav piav qhia vim li cas cov tshuaj nucleic acid tsim nyog tsim los ntawm plaub yam: kev ua lag luam thiab tshuaj xav tau, lub luag haujlwm tshwj xeeb thiab hloov tsis tau, kev ua tiav, thiab kev nyuaj.
Ua ntej tshaj plaws, muaj kev lag luam loj thiab cov tshuaj thov rau cov tshuaj nucleic acid. Raws li Lub Koom Haum Ntiaj Teb Kev Noj Qab Haus Huv (WHO), muaj ntau dua 7,000 tus kab mob tsis tshua muaj nyob hauv ntiaj teb, cuam tshuam txog 400 lab tus tib neeg. Kwv yees li 80% ntawm cov kab mob tsis tshua muaj tshwm sim los ntawm ib qho kev txawv txav ntawm cov noob, thiab li ntawm 95% ntawm lawv tsis muaj kev kho mob zoo. Cov neeg mob no muaj kev xav tau kev kho mob siab heev thiab muaj kev txaus siab them rau cov kev kho tshiab. Cov tshuaj Nucleic acid muaj qhov txiaj ntsig zoo hauv kev kho cov kab mob tsis tshua muaj vim lawv lub cev tshwj xeeb, tshuaj thiab tshuaj tshuaj. Lawv tuaj yeem tsom mus rau cov noob caj noob ces lossis cov khoom lag luam uas muaj qhov tshwj xeeb thiab kev sib raug zoo, thiab hloov kho lawv cov kev qhia lossis kev ua haujlwm ntawm kev thim rov qab thiab tswj tau. Lawv kuj tuaj yeem kov yeej qee qhov kev txwv ntawm cov tshuaj me me los yog cov tshuaj muaj protein, xws li bioavailability tsis zoo, tsis tshua muaj kev ruaj ntseg, immunogenicity, toxicity thiab phiv.
Thib ob, cov tshuaj nucleic acid muaj lub luag haujlwm tshwj xeeb thiab hloov tsis tau hauv kev kho noob. Gene therapy yog ib lub tswv yim zoo los kho cov kab mob los ntawm kev qhia, inhibiting lossis kho cov noob. Tam sim no, kev kho tshuaj nucleic acid tau muab faib ua peb yam tseem ceeb: qhia cov noob ib txwm los ntxiv cov haujlwm poob los ntawm kev hloov pauv noob, inhibiting lossis ntsiag to cov noob txawv txav los ntawm RNA cuam tshuam (RNAi), thiab ncaj qha kho cov kab mob txawv txav los ntawm kev kho cov noob (CRISPR-Cas9). Cov no yog txhua yam kev kho tshwj xeeb ntawm cov tshuaj nucleic acid uas tsis tuaj yeem ua tiav los ntawm lwm hom tshuaj. Piv txwv li, RNAi tuaj yeem xaiv qhov kev hais tawm ntawm tus kab mob- ua rau cov noob lossis cov proteins tsis cuam tshuam rau kev ua haujlwm ib txwm ntawm lwm cov noob lossis cov protein. Kev kho cov noob caj noob ces tuaj yeem hloov kho qhov DNA sib lawv liag ntawm cov noob caj noob ces thiab kho cov caj ces tsis xws luag.
Thib peb, muaj cov xwm txheej zoo ntawm cov tshuaj nucleic acid uas tau pom zoo rau kev lag luam lossis nyob rau hauv kev sim tshuaj. Piv txwv li, Onpattro (patisiran) yog thawj cov tshuaj RNAi tau pom zoo los ntawm US Food and Drug Administration (FDA) hauv 2018 rau kev kho mob ntawm cov kab mob transthyretin -mediated amyloidosis (hATTR), ib qho kab mob tsawg thiab tuag taus uas ua rau cov hlab ntsha puas thiab lub plawv tsis ua haujlwm. Onpattro tuaj yeem txo qhov tsim ntawm transthyretin (TTR) protein nyob rau hauv daim siab thiab tiv thaiv nws cov tsub zuj zuj ntawm cov ntaub so ntswg thiab lub cev. Lwm qhov piv txwv yog Luxturna (voretigene neparvovec), uas yog thawj cov tshuaj kho noob tau pom zoo los ntawm FDA hauv 2017 rau kev kho mob ntawm cov kab mob retinal dystrophy (IRD), uas tsis tshua muaj qhov muag uas ua rau dig muag. Luxturna tuaj yeem xa ib daim qauv ntawm RPE65 gene mus rau cov hlwb retinal thiab kho lawv lub peev xwm los tsim cov enzyme ua haujlwm uas tseem ceeb rau kev pom. Ntxiv mus, muaj ntau tshaj 300 nucleic acid tshuaj nyob rau hauv ntau theem ntawm kev kho mob kev loj hlob rau txawv indications, xws li mob cancer, kab mob, kab mob plawv, kab mob metabolic thiab neurological kab mob.
Thaum kawg, muaj qee yam kev nyuaj hauv kev tsim cov tshuaj nucleic acid, uas yuav pab txhawb kev txhim kho cov thev naus laus zis tshiab, txhim kho cov tshuaj zoo thiab ua tau zoo, thiab ua kom lub neej ntev ntawm cov tshuaj. Cov tshuaj Nucleic acid ntsib ntau yam teeb meem ntawm kev tsim, kev sib txuas, kev xa khoom, kev nyab xeeb thiab kev tswj hwm. Piv txwv li, nucleic acids tau yooj yim degraded los ntawm enzymes hauv vivo thiab muaj cov membrane permeability tsis zoo. Yog li ntawd, lawv yuav tsum tau hloov kho los yog encapsulated los ntawm ntau tus neeg nqa khoom los txhim kho lawv txoj kev ruaj ntseg thiab kev xa khoom zoo. Txawm li cas los xij, cov kev hloov kho lossis cov cab kuj tseem tuaj yeem cuam tshuam rau kev ua haujlwm lossis tshwj xeeb ntawm nucleic acids lossis ua rau muaj kev tiv thaiv kab mob lossis tshuaj lom. Yog li ntawd, nws yuav tsum muaj kev tsim kho tshiab tas li thiab kev ua kom zoo dua los kov yeej cov teeb meem no thiab ua tiav cov txiaj ntsig kho tau zoo.
Hauv kev xaus, cov tshuaj nucleic acid tsim nyog tsim vim lawv muaj peev xwm ua lag luam loj, cov txheej txheem kho mob tshwj xeeb, cov ntaub ntawv pov thawj thiab cov txheej txheem nyuaj. Lawv sawv cev rau thaj tsam tshiab ntawm biotechnology thiab tshuaj uas tuaj yeem ua rau muaj kev cia siab thiab muaj txiaj ntsig rau ntau lab tus neeg mob uas muaj ntau yam kab mob.
