Umda omkhulu weemolekyuli ezincinci--RNA ekujoliswe kuyo

2024-03-07 15:49:45

Kudala kucingwa ukuba ukusebenzisa imichiza ukulandelela i-RNA kulilize ngenxa yeempawu zayo eziguquguqukayo. Xa izazinzulu ziphumelela, kudla ngokuzenzekela.

Ngokomzekelo, kule minyaka ilishumi idluleyo, iMerck iye yafumanisa iyeza lokubulala iintsholongwane elisetyenziswayo elithintela uhlobo oluthile lwebhaktiriya ye-RNA. I-Pfizer yakhubeka ukuba ichiza layo linokuchaphazela ukuguqulelwa kweprotheyini elawula i-cholesterol.

Ngoku, iinkampani zamachiza zijonge ukwenza loo nto kanye. Ngoncedo lweendlela ezingcono zokulandelelana, iindlela zokuhlola, kunye nokuqonda ngokubanzi i-RNA, abaphandi banokubamba ngokulula ukubonakala kweemolekyuli zolwazi kunye nokuyila iziyobisi ezincamathela kuzo. Ukuba baphumelele, banokunceda ukufezekisa iithagethi zesifo ezinokuthi iimolekyuli ezincinci zifikelele ngoku, zibonelela ngeendlela ezintsha zokunyanga izifo ze-neurodeergenerative, umhlaza kunye nezinye iimeko. 18383aa279538923fe77cddddlkv

01 Zeziphi iimolekyuli ezincinci ezijolise kwi-RNA?

Iimolekyuli ezincinci ngamachiza asekelwe kwiikhemikhali ezijolise kwiiprotheyini, ukuthintela okanye ukuguqula indlela ezisebenza ngayo. Ngenxa yobukhulu bazo, iimolekyuli ezincinci zinokufikelela kuninzi lwezicubu emzimbeni womntu, zityibilike kwiiseli, kwaye zibophe kwindawo yeconcave kunye neconvex ekujoliswe kuyo.

Basisiseko seshishini loxubo-mayeza kwaye benza uninzi lwamachiza kwimarike. Kodwa umda weemolekyuli ezincinci ulinganiselwe. Kwimizila yemfuza ephantse ibe yi-20,000 eyaziwayo, emalunga nama-3,000 ijongwa njengenokutyiwa, nto leyo ethetha ukuba inokujoliswa kwiziyobisi. Kwiiprotheyini abazivelisayo, ngamakhulu ambalwa kuphela ekujoliswe kuwo kumachiza asele ekhona. Ezinye iiprotheyini ezininzi azinazo "iipokotho" apho iimolekyuli ezincinci zinokuzinzisa khona, nto leyo eyenza kube nzima ukufikelela kuzo.

Ukujolisa kwiimolekyuli ze-RNA ezinceda ukwenza iiproteni ezinxulumene nesifo kunokunika abenzi bamachiza enye indlela. Ngokwethiyori, iyeza linokuthintela ukuveliswa kweprotheyini enokuba yingozi okanye livelise iiproteni eziluncedo ngakumbi.

02 Encinci imolekyuli inzuzo eyahlukileyo yokujolisa kwi-RNA.

Iziyobisi eziphazamisana ne-RNA zibonakalise ukuba zizixhobo ezisebenzayo zokunyanga uluhlu lwezifo. Enye isuka kwi-Alnylam Pharmaceuticals, eyaqalisa indlela ebizwa ngokuba yi-RNA interference esebenzisa iimolekyuli ezincinci ze-RNA zokwenziwa ukuthulisa imfuza, ukubathintela ekuveliseni iiproteni eziyingozi.

Enye indlela efanayo, esetyenziswa yi-Ionis Pharmaceuticals kunye nezinye iinkampani, isebenzisa imicu ye-nucleic acid ebizwa ngokuba yi-antisense oligonucleotides ukulawula okanye ukucima ukuveliswa kweeprotheni. Zombini iinkampani ziye zaqhubela phambili uphando lwe-RNA, ngakumbi ijolise kumachiza ukunyanga izifo ezinqabileyo zofuzo ezifana ne-spinal muscular atrophy kunye ne-thyroid transamyloidosis, kunye neemeko eziqhelekileyo ezifana ne-cholesterol ephezulu. Imiphumo yeziyobisi inokuhlala iinyanga ngexesha.

Ngelixa inkqubela ebonakalayo yenziwe ekuhambiseni ezi zonyango ze-RNA emzimbeni, zisaxhomekeke kakhulu kwiithagethi zezifo esibindini, oku kuthetha ukuba kusekho izifo ezininzi ezingenako ukunyangwa.

03 Iimolekyuli ezincinci ezijolise kwi-RNA linyathelo elilandelayo.

Ngelixa inani lamachiza elakhiwe kwezinye iitekhnoloji ze-RNA zingenile kwimarike kwiminyaka yakutshanje, iimolekyuli ezincinci ezikwaziyo ukujolisa kwi-RNA ziqala ukubonisa amandla azo.

Owokuqala ukwenza njalo ngu-Evrysdi, ichiza eliphuhliswe ngokudibeneyo yi-Roche kunye ne-PTC Therapeutics kwaye ivunywe ngo-2020 ukunceda ukuvelisa iprotheni enqongopheleyo kwizigulane ezine-spinal muscular atrophy. Iyeza lovavanyo lesifo sika-Huntington elivela e-Novartis elibizwa ngokuba yi-branaplam likwakulingo lwabantu, kodwa imiba yokhuseleko yakutshanje inokuthintela inkqubela yalo.

Ukuza kuthi ga ngoku, akukho siqalo sokuphuhlisa iimolekyuli ezincinci ezijolise kwi-RNA ezithathe ichiza kulingo lwezonyango. Kodwa imibhobho yezinye iinkampani iqala ukumila. I-Skyhawk ibonakala ngathi ingaphambi kokungena kulingo lwezonyango, kwaye inkampani ibikhe yathi iceba ukuqalisa ukuhambisa iyeza layo lokuqala kulingo lwabantu ngaxesha lithile kulo nyaka.

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Umda omkhulu weemolekyuli ezincinci--RNA ekujoliswe kuyo