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Ubuvuzi bushya bwa Gene butanga ibyiringiro bishya kubarwayi ba selile na Thalassemia

BRL-101, yerekana iterambere ryibanze mu kuvura indwara zifata umuhoro (SCD) hifashishijwe ikoranabuhanga rya CRISPR / Cas9. Ubu buryo bushya bwo kuvura butanga ibyiringiro bishya byongera cyane urwego rwa hemoglobine (HbF), rushobora kuzamura cyane umusaruro w’abarwayi.

    Ubuvuzi bushya bwa Gene butanga ibyiringiro bishya kubarwayi ba selile na Thalassemia

    Guhindura Gene no kuvura thalassemia (12) Ishusho [24] .jpg Guhindura Gene no kuvura thalassemia 3Ishusho [24] .jpg

    Mu iterambere ryibanze ku barwayi barwaye indwara zifata umuhoro (SCD) na thalassemia, ubuvuzi bushya bwa gene bugaragaza intsinzi idasanzwe. Ubu buvuzi bukoresha ikoranabuhanga rigezweho rya CRISPR / Cas9 bwo guhindura gene, bwerekanye igipimo cyo gukira 100% mu bigeragezo by’amavuriro, bitanga ibyiringiro bishya ku barwanya izo ndwara zikomeye z’amaraso.

    Ubuvuzi, bwakozwe hamwe na porogaramu yihariye ya ModiHSC®, yibasira imizi ya geneti ya SCD na thalassemia. Muguhindura neza ibyongerera imbaraga BCL11A mumitsi ya autologique hematopoietic stem na progenitor selile, ubuvuzi butuma umubiri ubyara urwego rwinshi rwa hemoglobine (HbF). Urwego rwo hejuru rwa HbF rwerekanwe kurwanya ingaruka mbi ziterwa n'umuhoro hemoglobine (HbS) no kugabanya ibimenyetso bya SCD na thalassemia, harimo gukumira ibibazo biterwa na vaso no kugabanya amaraso make.

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    BIOOCUS, imbaraga zikomeye mu bijyanye no kuvura gene, ku bufatanye n’ibitaro bya Lu Daopei, yagize uruhare runini mu kugeza ubu buvuzi bushya ku barwayi. 

    Intsinzi yo kuvura ivuriro ntagereranywa, abarwayi 15 bavuwe kugeza ubu, bose bagera ku gukira burundu ndetse no kuzamura imibereho yabo. Iki gipimo cyo gukiza 100% nintambwe ikomeye mukurwanya SCD na thalassemia.

     

    Ubuvuzi bumaze kumenyekana ku rwego mpuzamahanga, hamwe n’inzobere ku isi hose bavuga ko ari intambwe yo kuvura indwara z’amaraso. Ntabwo igaragara gusa kubikorwa byayo gusa ahubwo no mubikorwa byayo. Bitandukanye nubundi buryo bwo kuvura gene, bushobora kuba buhenze cyane, ubu buvuzi butanga uburyo bworoshye bwo kugena ibiciro, bigatuma buba amahitamo meza kubarwayi benshi.

    Mu rubanza rumwe rukomeye, umurwayi wimyaka 12 ufite ibibazo bya vaso-occlusive hamwe na anemia ikabije ya hemolitike yahuye nibimenyetso burundu nyuma yo kuvurwa nubuvuzi bwa gene. Uru rubanza, hamwe n’abandi, rugaragaza ubushobozi bwo guhindura imiti ya SCD na thalassemia ku isi yose.

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    Mugihe BIOOCUS ikomeje kwagura uburyo bwo kuvura, harimo n’ibizamini by’amavuriro biri imbere mu Bushinwa, ejo hazaza hasa n’icyizere ku bahuye n’izi ndwara zitoroshye. Ku nkunga y'ibitaro bya Lu Daopei, ubu buvuzi bugiye gusobanura neza urwego rwo kwita kuri SCD na thalassemia, rutanga ubuzima bushya ku buzima bw'abarwayi batabarika.

    Niba wowe cyangwa uwo ukunda barwaye indwara zifata umuhoro cyangwa thalassemia kandi ukaba ushishikajwe no gushakisha ubu buvuzi bushya, turagutumiye kutwandikira. Ikipe yacu yiteguye kuguha inkunga namakuru ukeneye kugirango ufate ibyemezo byuzuye kubyerekeye urugendo rwawe rwubuzima.