He Huarahi Hou te Whakaora i te Mate Pūtau Hāri me te Thalassemia nā te Whakaora Ira Hou

He whakatutukitanga nui tēnei mā te hapori hauora me ngā tūroro e whawhai ana ki te mate toto toto (SCD) me te thalassemia, ā, kua whakaatuhia e te rongoā ira matatau e whakamahi ana i te hangarau CRISPR/Cas9 he tere whakaora whakamiharo 100% i roto i ngā whakamātautau haumanu. Ka whakaratohia e tēnei rongoā hou he tumanako hou ki ngā miriona tūroro e pāngia ana e ēnei mate toto ngoikore, i mua i hiahiatia he whakawhiti toto mō te wā katoa me ngā kōwhiringa maimoatanga iti.

Ko tētahi tauira rongonui o tēnei whanaketanga ko te take o tētahi tauira whare wānanga 21-tau te pakeke nō Guilin, Haina, i kitea he mate beta-thalassemia kino i te waru marama noa iho o tōna pakeke. Neke atu i te rua tekau tau, i whakawhirinaki tōna oranga ki ngā whakawhiti toto auau hei whakahaere i tōna āhua. Heoi, nā te whakaurunga mai o tētahi rongoā ira matatau, i whakawhanakehia mā te whakamahi i te tūāpapa ModiHSC® motuhake, i huri ai tōna ara.

I te 8 o Hakihea, 2022, i whiwhi te tūroro i ngā pūtau take hematopoietic i whakatikatikaina e te ira, i aro tika ki te whakarerekētanga ira i puta ai tōna āhua. I roto i ngā marama torutoru i muri mai, ka hoki ōna tohu toto, tae atu ki ngā toto whero, ngā toto mā, me ngā pereti toto, ki ngā taumata noa. I te 17 o Pēpuere, 2023, kua mana te tūroro ki te kore e hiahia ki te whakawhiti toto, e tohu ana i te whakaoranga katoa me te upoko hou o tōna ao.

Ka mahi tēnei rongoā mā te whakatika i te whakarei ake BCL11A i roto i ngā pūtau take hematopoietic me ngā pūtau tupuna o te tūroro, e āhei ai te whakaputa i ngā taumata teitei ake o te hemoglobin fetal (HbF). Ka āwhina te HbF teitei ki te ārai i ngā pānga kino o te hemoglobin sickle (HbS) i roto i ngā tūroro SCD, ā, ka whakaiti i ngā tohu i roto i te SCD me te thalassemia, tae atu ki te aukati i ngā raruraru vaso-occlusive me te whakamamae i te anemia hemolytic.

Hei tūroro pakeke tuatahi i roto i tēnei whakamātautau haumanu, he tohu tumanako tēnei mō ētahi atu tūroro pakeke e pāngia ana e te thalassemia kino, he tokomaha o rātou kua ngaro te whai wāhi ki te rongoā mā te whakawhitinga pūtau take tuku iho nā te korenga o tētahi kaituku toto e ōrite ana. Ko te angitu o tēnei maimoatanga e whakaatu ana i te kaha o te rongoā ira ki te huri i te whakahaere i ngā mate toto ira me te kawe mai i ngā otinga whakaora mō te wā roa ki ngā tūroro puta noa i te ao.

Ko tēnei tauira paionia e tohu ana i tētahi panonitanga tauira o te huarahi e whakatata atu ai tātou me te whakahaere i ēnei āhuatanga, e tuku ana i te āheinga o tētahi oranga ki tua atu i te oranga tonutanga—he oranga e ki ana i ngā whai wāhitanga me te kupu whakaari mō tētahi heke mai pai ake.