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Unyango lweGene olutsha lubonelela ngeThemba elitsha kwiSickle Cell kunye nezigulana zeThalassemia

I-BRL-101, imele ukuqhubela phambili okusemgangathweni kunyango lwe-sickle cell disease (SCD) ngokusebenzisa i-CRISPR/Cas9 iteknoloji yokuhlela i-gene. Olu nyango lunenguqulelo entsha lunika ithemba elitsha ngokunyusa kakhulu amanqanaba e-fetal hemoglobin (HbF), enokuphucula kakhulu iziphumo zesigulana.

    Unyango lweGene olutsha lubonelela ngeThemba elitsha kwiSickle Cell kunye nezigulana zeThalassemia

    Ukuhlelwa kofuzo kunye nonyango lwe-thalassemia (12) Umfanekiso[24].jpg Ukuhlelwa kwemfuza kunye nonyango lwe-thalassemia 3Image[24].jpg

    Kuphuhliso olusisiseko kwizigulana ezinesifo se-sickle cell (SCD) kunye ne-thalassemia, unyango olutsha lwemfuza lubonisa impumelelo emangalisayo. Olu nyango, olusebenzisa itekhnoloji yokuhlela yofuzo ye-CRISPR/Cas9 ephucukileyo, ibonise i-100% yonyango lonyango kulingo lwezonyango, inika ithemba elihlaziyiweyo kwabo balwa nezi ngxaki zinzima zegazi.

    Unyango, oluphuhliswe kunye neqonga le-ModiHSC® lobunikazi, lijolise kwiingcambu zofuzo ze-SCD kunye ne-thalassemia. Ngokuhlela ngokuchanekileyo isixhasi se-BCL11A kwisiqu esine-autologous hematopoietic kunye neeseli zeprogenitor, unyango lwenza ukuba umzimba uvelise amanqanaba aphezulu e-fetal hemoglobin (HbF). Amanqanaba e-HbF aphakanyisiweyo abonakaliswe ukuchasana nemiphumo eyingozi ye-sickle hemoglobin (HbS) kunye nokunciphisa iimpawu ze-SCD kunye ne-thalassemia, kubandakanywa nokuthintela iingxaki ze-vaso-occlusive kunye nokunciphisa i-anemia ye-hemolytic.

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    I-BIOOCUS, ibutho elikhokelayo kwinkalo yonyango lwemfuza, ngokubambisana neSibhedlele saseLu Daopei, ibe negalelo ekuziseni olu nyango lutsha kwizigulana. 

    Impumelelo yeklinikhi yonyango ayinakuthelekiswa nanto, kunye nezigulana ezili-15 ezinyangwayo ukuza kuthi ga ngoku, zonke zifumana uxolelo olupheleleyo kunye nophuculo olubonakalayo kumgangatho wobomi babo. Eli nqanaba le-100% lokunyanga linyathelo elibalulekileyo kumlo ochasene ne-SCD kunye ne-thalassemia.

     

    Olu nyango lufumene ukuqondwa kumazwe ngamazwe, neengcali kwihlabathi jikelele ziluncoma njengempumelelo kunyango lwezifo zegazi zemfuzo. Ibalasele kungekuphela nje ekusebenzeni kwayo kodwa nakwindleko zayo ezifanelekileyo. Ngokungafaniyo nolunye unyango lwemfuza, olunokubiza kakhulu, olu nyango lubonelela ngemodeli yexabiso efikeleleka ngakumbi, ilwenza ibe lukhetho olusebenzayo kuluhlu olubanzi lwezigulane.

    Kwimeko enye enyanzelisayo, isigulane esineminyaka eyi-12 esineengxaki eziphindaphindiweyo ze-vaso-occlusive kunye ne-anemia enzima ye-hemolytic yafumana ukupheliswa okupheleleyo kweempawu ezilandela unyango ngolu nyango lwemfuza. Eli tyala, phakathi kwezinye, liqaqambisa amandla enguqu kunyango lwe-SCD kunye nezigulana zethalassemia kwihlabathi liphela.

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    Njengoko i-BIOOCUS iqhubeka nokwandisa ukufumaneka kolu nyango, kubandakanywa nolingo lwezonyango oluzayo e-China, ikamva lijongeka lithembisa abo bachatshazelwe zezi zifo zingumngeni. Ngenkxaso yeSibhedlele i-Lu Daopei, olu nyango lusetelwe ukuchaza ngokutsha umgangatho wokhathalelo lwe-SCD kunye ne-thalassemia, lunikezela ngengqeshiso entsha yobomi kwizigulana ezingenakubalwa.

    Ukuba wena okanye umntu omthandayo uchatshazelwa sisifo se-sickle cell okanye ithalassemia kwaye unomdla wokuphonononga olu nyango lunenguqulelo entsha, siyakumema ukuba uqhagamshelane nathi. Iqela lethu likulungele ukukunika inkxaso kunye nolwazi oludingayo ukuze wenze izigqibo ezinolwazi malunga nohambo lwakho lwezempilo.