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Innovative Gene Therapy Yana Bada Sabon Fata Ga Marasa Lafiyar Sickle Cell da Thalassemia

BRL-101, yana wakiltar ci gaba mai ban sha'awa a cikin maganin cutar sikila (SCD) ta hanyar fasahar gyara kwayoyin halittar CRISPR/Cas9. Wannan sabuwar fasahar tana ba da sabon bege ta hanyar haɓaka matakan haemoglobin na tayin (HbF), wanda zai iya haɓaka sakamakon haƙuri sosai.

    Innovative Gene Therapy Yana Bada Sabon Fata Ga Marasa Lafiyar Sickle Cell da Thalassemia

    Gyaran Halittu da maganin thalassemia (12) Hoto[24].jpg Gyaran Halittu da maganin thalassemia 3Image[24].jpg

    A cikin ci gaba mai ban sha'awa ga marasa lafiya masu fama da cutar sikila (SCD) da thalassemia, sabon maganin kwayoyin halitta yana nuna babban nasara. Wannan jiyya, wanda ke amfani da fasahar gyaran gyare-gyare na CRISPR/Cas9 na ci gaba, ya nuna adadin warkewa 100% a cikin gwaje-gwajen asibiti, yana ba da sabon bege ga waɗanda ke fama da waɗannan munanan cututtukan jini.

    Maganin, wanda aka haɓaka tare da dandamali na ModiHSC® na mallakar mallaka, yana hari akan tushen kwayoyin halittar SCD da thalassaemia. Ta hanyar gyara mai haɓaka BCL11A daidai a cikin tushe na hematopoietic autologous da sel masu zuwa, maganin yana bawa jiki damar samar da matakan haemoglobin na tayin (HbF). An nuna haɓakar matakan HbF don magance lahani na haemoglobin na sikila (HbS) da rage alamun SCD da thalassaemia, gami da rigakafin rikice-rikice na vaso-occlusive da rage yawan anemia na hemolytic.

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    BOOOCUS, babbar jami'a a fannin nazarin kwayoyin halitta, tare da hadin gwiwar asibitin Lu Daopei, sun taka rawar gani wajen kawo wannan sabuwar magani ga marasa lafiya. 

    Nasarar asibitin ba ta misaltuwa, inda aka yi wa majinyata 15 magani kawo yanzu, duk sun sami cikakkiyar gafara da kuma inganta rayuwar su. Wannan kashi 100% na magani wani muhimmin ci gaba ne a yaƙin SCD da thalassaemia.

     

    Maganin ya sami karɓuwa a duniya, inda masana a duk faɗin duniya suka yaba da shi a matsayin wani ci gaba a cikin maganin cututtukan jini. Ya fice ba kawai don ingancinsa ba har ma don ƙimar sa. Ba kamar sauran hanyoyin kwantar da hankali ba, waɗanda za su iya yin tsada mai tsada, wannan magani yana ba da samfurin farashi mai sauƙi, yana mai da shi zaɓi mai dacewa ga ɗimbin marasa lafiya.

    A cikin wani yanayi mai tursasawa, mai shekaru 12 mai haƙuri tare da rikice-rikice na vaso-occlusive da ke faruwa da kuma anemia mai tsanani na hemolytic ya sami cikakkiyar ƙarewar bayyanar cututtuka bayan jiyya tare da wannan maganin kwayoyin halitta. Wannan shari'ar, da sauransu, tana ba da haske game da yuwuwar canji na jiyya ga SCD da marasa lafiya na thalassemia a duk duniya.

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    Yayin da BOOOCUS ke ci gaba da fadada samar da wannan maganin, gami da gwaje-gwajen asibiti da ke tafe a kasar Sin, nan gaba na da kyau ga wadanda suka kamu da wadannan cututtuka masu kalubale. Tare da goyan bayan Asibitin Lu Daopei, an saita wannan maganin don sake fayyace ma'aunin kula da SCD da thalassaemia, yana ba da sabon hayar rayuwa ga marasa lafiya marasa adadi.

    Idan kai ko masoyi yana fama da cutar sikila ko thalassaemia kuma kuna sha'awar bincika wannan sabuwar magani, muna gayyatar ku don tuntuɓar mu. Ƙungiyarmu a shirye take don ba ku goyon baya da bayanan da kuke buƙata don yanke shawara mai zurfi game da tafiyar lafiyar ku.