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Innovative Gene Therapy Imapereka Chiyembekezo Chatsopano kwa Odwala Sickle Cell ndi Thalassemia

BRL-101, ikuyimira kupita patsogolo kwakukulu pamankhwala a sickle cell disease (SCD) kudzera muukadaulo wa CRISPR/Cas9 wosintha ma gene. Thandizo lamakonoli limapereka chiyembekezo chatsopano mwa kuonjezera kwambiri mlingo wa hemoglobin wa fetal (HbF), womwe ukhoza kusintha kwambiri zotsatira za odwala.

    Innovative Gene Therapy Imapereka Chiyembekezo Chatsopano kwa Odwala Sickle Cell ndi Thalassemia

    Kusintha kwa majini ndi chithandizo cha thalassemia (12) Image[24].jpg Kusintha kwa majini ndi chithandizo cha thalassemia 3Image[24].jpg

    Pachitukuko chachikulu cha odwala omwe ali ndi matenda a sickle cell (SCD) ndi thalassemia, chithandizo chatsopano cha majini chikuwonetsa kupambana kodabwitsa. Chithandizochi, chomwe chimagwiritsa ntchito ukadaulo wapamwamba wosintha ma gene CRISPR/Cas9, awonetsa chiwopsezo cha 100% pamayesero azachipatala, ndikupereka chiyembekezo chatsopano kwa omwe akulimbana ndi matenda oopsawa.

    Thandizo, lopangidwa ndi nsanja ya ModiHSC®, limayang'ana mizu ya SCD ndi thalassemia. Pokonza bwino chowonjezera cha BCL11A mu tsinde la autologous hematopoietic stem and progenitor cell, chithandizochi chimathandiza kuti thupi lipange kuchuluka kwa fetal hemoglobin (HbF). Miyezo yokwera ya HbF yasonyezedwa kuti ikulimbana ndi zotsatira zowononga za sickle hemoglobin (HbS) ndi kuchepetsa zizindikiro za SCD ndi thalassemia, kuphatikizapo kupewa matenda a vaso-occlusive ndi kuchepetsa kuchepa kwa magazi m'thupi.

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    BIOOCUS, yemwe ndi wotsogola pantchito ya gene therapy, mogwirizana ndi Chipatala cha Lu Daopei, athandizira kwambiri kubweretsa chithandizo chamakono kwa odwala. 

    Kupambana kwachipatala kwachipatala sikungafanane, ndi odwala 15 omwe adalandira chithandizo mpaka pano, onse akulandira chikhululukiro chathunthu komanso kusintha kwakukulu kwa moyo wawo. Kuchiritsa kwa 100% kumeneku ndi gawo lofunikira kwambiri polimbana ndi SCD ndi thalassemia.

     

    Thandizoli ladziwika padziko lonse lapansi, ndipo akatswiri padziko lonse lapansi amaliyamikira ngati njira yothandiza kwambiri pochiza matenda obwera chifukwa cha majini. Sichidziwika kokha chifukwa cha mphamvu zake komanso chifukwa cha mtengo wake. Mosiyana ndi mankhwala ena amtundu, omwe amatha kukhala okwera mtengo kwambiri, chithandizochi chimapereka njira yofikira pamitengo, zomwe zimapangitsa kukhala njira yabwino kwa odwala ambiri.

    Pankhani ina yochititsa chidwi, wodwala wazaka 12 yemwe anali ndi vuto la vaso-occlusive mobwerezabwereza komanso kuchepa kwa magazi m'thupi la hemolytic kutha kwa zizindikiro pambuyo pa chithandizo cha jini. Mlanduwu, pakati pa ena, ukuwonetsa kuthekera kosinthika kwa chithandizo cha odwala a SCD ndi thalassemia padziko lonse lapansi.

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    Pamene BIOOCUS ikupitiliza kukulitsa kupezeka kwa mankhwalawa, kuphatikiza mayeso azachipatala omwe akubwera ku China, tsogolo likuwoneka ngati labwino kwa omwe akhudzidwa ndi matenda ovutawa. Mothandizidwa ndi Chipatala cha Lu Daopei, chithandizochi chakhazikitsidwa kuti chifotokozenso mulingo wa chisamaliro cha SCD ndi thalassemia, kupereka moyo watsopano kwa odwala osawerengeka.

    Ngati inu kapena okondedwa anu akukhudzidwa ndi matenda a sickle cell kapena thalassemia ndipo mukufuna kudziwa chithandizo chamakonochi, tikukupemphani kuti mutilankhule. Gulu lathu lakonzeka kukupatsirani chithandizo ndi chidziwitso chomwe mukufuna kuti mupange zisankho zodziwika bwino zaulendo wanu wazachipatala.