Thalassemia Chen Yan

Mukubudirira kukuru kwenharaunda yezvekurapa nevarwere vari kurwisana nechirwere chesickle cell (SCD) nethalassemia, kurapwa kwemajini epamusoro kunoshandisa tekinoroji yeCRISPR/Cas9 kwakaratidza mwero unoshamisa wekurapa 100% mukuyedzwa kwekiriniki. Kurapa uku kunoshamisa kunopa tariro itsva kumamiriyoni evarwere vanotambura nezvirwere zveropa izvi zvinoremadza, izvo zvaimboda kuwedzerwa ropa kwehupenyu hwese uye sarudzo shoma dzekurapa.

Muenzaniso unoshamisa wekubudirira uku ndewemudzidzi wepayunivhesiti ane makore makumi maviri nerimwe wekuGuilin, China, uyo akaonekwa aine chirwere che beta-thalassemia chakanyanya aine mwedzi misere chete. Kwemakore anopfuura makumi maviri, hupenyu hwake hwaitsamira pakuwedzerwa ropa nguva nenguva kuti ararame nechirwere chake. Zvisinei, kuunzwa kwekurapa kwemajini epamusoro, kwakagadzirwa uchishandisa puratifomu yeModiHSC®, kwakachinja nzira yake.

Musi wa8 Zvita 2022, murwere akagamuchira masero eropa akagadziriswa nemajini ayo akanyatsonanga nekugadzirisa kushanduka kwemajini kwakakonzera chirwere chake. Mumwedzi mishoma yakatevera, zviratidzo zveropa rake, zvinosanganisira masero matsvuku eropa, masero machena eropa, uye maplatelet, zvakadzokera padanho rakajairika. Pakazosvika Kukadzi 17, 2023, murwere akanga asisadi kuwedzerwa ropa, zvichiratidza kurapwa kwakakwana uye chitsauko chitsva muhupenyu hwake.

Kurapa uku kunoshanda nekugadzirisa BCL11A enhancer mumasero eropa emurwere uye emuviri wake, izvo zvinoita kuti hemoglobin yemwana (HbF) iwedzere. HbF yakakwira inobatsira kurwisa migumisiro inokuvadza ye sickle hemoglobin (HbS) muvarwere veSCD uye inoderedza zviratidzo muSCD nethalassemia, kusanganisira kudzivirira matambudziko eropa nekuderedza ropa nekuderedza anemia.

Semurwere mukuru wekutanga muongororo iyi yekiriniki, nyaya yake inopa tariro kune vamwe varwere vakuru vane thalassemia yakanyanya, vazhinji vavo vakarasikirwa nemukana wekurapwa kuburikidza nekuisirwa masero emuviri nekuda kwekushaikwa kwemupi anoenderana nawo. Kubudirira kwekurapwa uku kunoratidza mukana wekurapa majini ekuchinja kurapa kwezvirwere zveropa zvemajini uye kuunza mhinduro dzenguva refu, dzinorapa kuvarwere pasi rese.

Nyaya iyi yekutanga inomiririra shanduko huru munzira yatinotarisana nayo uye yatinobata nayo mamiriro ezvinhu aya, ichipa mukana wehupenyu hunopfuura kurarama—huzere nemikana uye vimbiso yeramangwana riri nani.