Sabuwar Maganin Halitta Ya Samu Nasara Wajen Magance Cututtukan Sickle Cell da Thalassemia

A wani gagarumin ci gaba ga al'ummar likitoci da marasa lafiya da ke fama da cutar sikila (SCD) da thalassemia, wani ci gaba na maganin kwayoyin halitta da ke amfani da fasahar CRISPR/Cas9 ya nuna kyakkyawan sakamako 100% na warkewa a gwaje-gwajen asibiti. Wannan sabon maganin yana ba da sabon bege ga miliyoyin marasa lafiya da ke fama da waɗannan cututtukan jini masu rauni, waɗanda a da suka buƙaci a yi musu ƙarin jini tsawon rai da kuma zaɓuɓɓukan magani kaɗan.

Wani misali mai kyau na wannan ci gaba shine batun wani ɗalibi ɗan shekara 21 daga jami'a daga Guilin, China, wanda aka gano yana da cutar beta-thalassemia mai tsanani yana da watanni takwas kacal. Tsawon shekaru sama da ashirin, rayuwarsa ta dogara ne akan yawan zubar jini don kula da lafiyarsa. Duk da haka, gabatar da wani sabon maganin kwayoyin halitta, wanda aka haɓaka ta amfani da dandamalin ModiHSC® na musamman, ya canza yanayinsa.

A ranar 8 ga Disamba, 2022, majinyacin ya karɓi ƙwayoyin halittar jini da aka gyara ta hanyar kwayoyin halitta waɗanda suka yi daidai da kuma gyara canjin halittar da ke haifar da yanayinsa. A cikin 'yan watanni masu zuwa, alamun jininsa, waɗanda suka haɗa da ƙwayoyin jinin ja, ƙwayoyin jinin fari, da ƙwayoyin platelets, sun koma matsayinsu na yau da kullun. Zuwa ranar 17 ga Fabrairu, 2023, majinyacin ya zama ba shi da alaƙa da ƙarin jini, wanda hakan ke nuna cikakken waraka da sabon babi a rayuwarsa.

Wannan maganin yana aiki ta hanyar gyara sinadarin BCL11A a cikin ƙwayoyin jinin majiyyaci da kuma ƙwayoyin halittar jini, wanda ke ba da damar samar da ƙarin matakan haemoglobin na tayi (HbF). Ƙara yawan HbF yana taimakawa wajen magance illolin da sickle hemoglobin (HbS) ke haifarwa ga marasa lafiya da SCD kuma yana rage alamun cutar SCD da thalassemia, gami da hana rikice-rikicen vaso-occlusive da rage yawan jinin hemolytic.

A matsayinsa na babban majiyyaci na farko a cikin wannan gwajin asibiti, lamarinsa ya ba da bege ga sauran manyan marasa lafiya da ke fama da mummunar cutar thalassemia, waɗanda da yawa daga cikinsu sun rasa damar samun waraka ta hanyar dashen ƙwayoyin halitta na gargajiya saboda rashin samun mai ba da gudummawa mai dacewa. Nasarar wannan maganin ta nuna yuwuwar maganin kwayoyin halitta don kawo sauyi ga kula da cututtukan jini na kwayoyin halitta da kuma kawo mafita na dogon lokaci ga marasa lafiya a duk duniya.

Wannan shari'ar ta farko tana wakiltar wani sauyi a cikin yadda muke tunkarar waɗannan yanayi da kuma magance su, wanda ke ba da damar rayuwa fiye da rayuwa—wadda ke cike da dama da alƙawarin kyakkyawar makoma.