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Innovative Gene Therapy Inopa Tariro Itsva yeSickle Cell uye Thalassemia Varwere

BRL-101, inomiririra kufambira mberi kwepasi mukurapa kwechirwere chegomarara (SCD) kuburikidza neCRISPR/Cas9 gene editing tekinoroji. Kurapa kutsva uku kunopa tariro itsva nekuwedzera zvakanyanya fetal hemoglobin (HbF) mazinga, ayo anogona kuvandudza zvakanyanya mhedzisiro yemurwere.

    Innovative Gene Therapy Inopa Tariro Itsva yeSickle Cell uye Thalassemia Varwere

    Gene editing uye thalassemia kurapwa (12) Mufananidzo[24].jpg Gene editing uye thalassemia kurapwa 3Image[24].jpg

    Mukukura kunokatyamadza kwevarwere vanorwara nechirwere chesickle cell (SCD) uye thalassemia, mushonga mutsva wejini uri kuratidza budiriro inoshamisa. Mushonga uyu, unoshandisa tekinoroji yemhando yepamusoro yeCRISPR/Cas9, yakaratidza chiyero chekurapa chegumi muzana muzviyedzo zvekiriniki, ichipa tariro itsva kune avo vari kurwisa zvirwere zvakanyanya zveropa.

    The therapy, yakagadziridzwa pamwe nevaridzi veModiHSC® papuratifomu, inotarisa midzi yeSCD uye thalassemia. Nekunyatso gadzirisa iyo BCL11A enhancer mu autologous hematopoietic stem uye maseru ekutanga, kurapa kunoita kuti muviri ugadzire huwandu hwepamusoro hwe fetal hemoglobin (HbF). Yakakwira HbF mazinga akaratidzwa kurwisa zvinokuvadza zvesickle hemoglobin (HbS) uye kuderedza zviratidzo zveSCD uye thalassemia, kusanganisira kudzivirira kwevaso-occlusive matambudziko uye kuderedza hemolytic anemia.

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    BIOOCUS, simba rinotungamira mumunda wegene therapy, mukubatana neLu Daopei Hospital, yakabatsira mukuunza kurapwa uku kuvarwere. 

    Kubudirira kwekurapa kwekurapa hakuenzaniswi, nevarwere gumi nevashanu vakarapwa kusvika pari zvino, vese vachiwana kuregererwa kwakakwana uye kuvandudzwa kwakakosha muhupenyu hwavo. Iyi 100% yekurapa chiyero chiitiko chakakosha mukurwisa SCD uye thalassemia.

     

    Kurapa uku kwave kucherechedzwa pasi rose, nenyanzvi pasi rose vachikurumbidza sebudiriro mukurapa kwekusagadzikana kweropa. Iyo inomira kunze kwete nekuda kwekuita kwayo chete asiwo kune yayo mutengo-inoshanda. Kusiyana nemamwe marapirwo emajini, ayo anogona kudhura zvakanyanya, kurapwa uku kunopa modhi yemitengo inowanikwa, zvichiita kuti ive sarudzo inogoneka kune huwandu hwakawanda hwevarwere.

    Mune imwe nyaya inomanikidza, murwere ane makore gumi nemaviri ane zvinowanzoitika vaso-occlusive crises uye yakanyanya hemolytic anemia akawana kupera kwakakwana kwezviratidzo zvichitevera kurapwa neiyi gene therapy. Mhosva iyi, pakati pevamwe, inosimbisa mukana wekushandura wekurapa kwevarwere veSCD uye thalassemia pasi rese.

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    Sezvo BIOOCUS ichiramba ichiwedzera kuwanikwa kwekurapa uku, kusanganisira kuedzwa kwekiriniki kuri kuuya kuChina, ramangwana rinotaridzika kune avo vanobatwa nezvirwere izvi zvakaomarara. Nerutsigiro rwechipatara cheLu Daopei, kurapa uku kwakaiswa kuti kutsanangure chiyero chekuchengeta SCD uye thalassemia, ichipa hupenyu hutsva hwehupenyu hwevarwere vasingaverengeki.

    Kana iwe kana mudikanwi akabatwa nechirwere chesickle cell kana thalassemia uye muchifarira kuongorora kurapa kutsva uku, tinokukokai kuti mutibate. Chikwata chedu chakagadzirira kukupa rutsigiro uye ruzivo rwaunoda kuti uite sarudzo dzine ruzivo nezverwendo rwako rwehutano.