01020304
I-Big Frontier yama-molecule amancane--i-RNA eqondiswe
2024-03-07 15:49:45
Sekuyisikhathi eside kucatshangwa ukuthi ukusebenzisa amakhemikhali ukulandelela i-RNA kwakuyize ngenxa yezinto zayo eziguquguqukayo. Lapho ososayensi bephumelela, ngokuvamile kwenzeka ngephutha.
Ngokwesibonelo, eminyakeni eyishumi edlule, i-Merck ithole isibulala-magciwane esihlolwayo esakhiwayo esivimba uhlobo lwe-RNA yebhaktheriya. I-Pfizer yakhubeka ngokuthi umuthi wayo ungathinta ukuhunyushwa kwephrotheni elawula i-cholesterol.
Manje, izinkampani zezidakamizwa zihlose ukwenza lokho kanye. Ngosizo lwezindlela ezingcono zokulandelanisa, izindlela zokuhlola, kanye nokuqonda okubanzi kwe-RNA, abacwaningi bangakwazi ukuthwebula kalula ukubukeka kwama-molecule olwazi futhi baklame izidakamizwa ezinamathela kuwo. Uma bephumelela, bangasiza ekufinyeleleni okuhlosiwe kwezifo ama-molecule amancane angeke akwazi ukuwafinyelela njengamanje, anikeze izindlela ezintsha zokwelapha izifo ze-neurodeergenerative, umdlavuza nezinye izimo.
01 Imaphi ama-molecule amancane aqondise i-RNA?
Ziyisisekelo semboni yezemithi futhi zakha ingxenye enkulu yezidakamizwa emakethe. Kodwa ububanzi bama-molecule amancane bulinganiselwe. Ezakhini zofuzo ezicishe zibe ngu-20,000 ezaziwayo, ezingaba ngu-3,000 zithathwa njengezingasebenziseka, okusho ukuthi zingahlaselwa ngezidakamizwa. Kumaprotheni abawakhiqizayo, amakhulu ambalwa kuphela okuhloswe ngawo emithini ekhona. Amanye amaprotheni amaningi awanawo "amaphakethe" lapho ama-molecule amancane angashumeka khona, okwenza kube nzima ukufinyelela kuwo.
Ukukhomba ama-molecule e-RNA asiza ukwenza amaprotheni ahlobene nezifo kunganikeza abenzi bezidakamizwa enye indlela. Ngokombono, umuthi ungavimba ukukhiqizwa kwephrotheni engaba yingozi noma ukhiqize amaprotheni anenzuzo eyengeziwe.
Enye indlela efanayo, esetshenziswa i-Ionis Pharmaceuticals nezinye izinkampani, isebenzisa imicu ye-nucleic acid ebizwa ngokuthi i-antisense oligonucleotides ukuze ilawule noma ivale ukukhiqizwa kwamaprotheni. Zombili izinkampani zenze ucwaningo oluthuthukisiwe lwe-RNA, ikakhulukazi eziqondise emithini yokwelapha izifo zofuzo ezingavamile njenge-spinal muscular atrophy kanye ne-thyroid transamyloidosis, kanye nezimo ezivame kakhulu ezifana ne-cholesterol ephezulu. Imiphumela yomuthi ingahlala izinyanga ngesikhathi.
Nakuba sekwenziwe inqubekelaphambili ebalulekile ekuletheni lezi zindlela zokwelapha i-RNA emzimbeni, zisanqunyelwe kakhulu ezihlotsheni zezifo esibindini, okusho ukuthi kusenezifo eziningi ezingelapheka.
03 Ama-molecule amancane aqondise i-RNA ayisinyathelo esilandelayo.
Owokuqala ukwenza kanjalo kwaba yi-Evrysdi, umuthi owakhiwe ngokuhlanganyela yi-Roche kanye ne-PTC Therapeutics futhi wagunyazwa ngo-2020 ukusiza ukukhiqiza amaprotheni ashodayo ezigulini ezine-spinal muscular atrophy. Umuthi wokuhlola wesifo sika-Huntington ovela e-Novartis obizwa ngokuthi i-branaplam nawo usezivivinyweni zabantu, kodwa izinkinga zakamuva zokuphepha zingathiya ukuqhubeka kwawo.
Kuze kube manje, akukho siqalo esakha ama-molecule amancane aqondise i-RNA okuthathe umuthi ezivivinyweni zomtholampilo. Kepha amapayipi ezinkampani ezithile aseqala ukwakheka. I-Skyhawk ibonakala ingaphambi kokungena ezivivinyweni zomtholampilo, futhi inkampani phambilini ithe ihlela ukuqala ukuhambisa umuthi wayo wokuqala ekuhlolweni kwabantu kwesinye isikhathi kulo nyaka.