Unyango lweZini ezintsha lubonelela ngethemba elitsha kwizigulane ezineSickle Cell kunye neThalassemia

Unyango lweZini ezintsha lubonelela ngethemba elitsha kwizigulane ezineSickle Cell kunye neThalassemia

Kwinkqubela phambili entsha kwizigulane ezinesifo se-sickle cell (SCD) kunye ne-thalassemia, unyango olutsha lwe-gene lubonisa impumelelo emangalisayo. Olu nyango, olusebenzisa ubuchwepheshe obuphambili bokuhlela i-gene be-CRISPR/Cas9, lubonise izinga lokunyanga eliyi-100% kwiimvavanyo zeklinikhi, lunika ithemba elitsha kwabo balwa nezi ngxaki zinzima zegazi.

Olu nyango, oluphuhliswe ngeqonga leModiHSC® eliyipropathi, lujolise kwiingcambu zemfuza ze-SCD kunye ne-thalassemia. Ngokuhlela ngokuchanekileyo i-BCL11A enhancer kwiiseli ze-hematopoietic stem kunye ne-progenitor, olu nyango lwenza umzimba ukwazi ukuvelisa amanqanaba aphezulu e-fetal hemoglobin (HbF). Amanqanaba aphezulu e-HbF abonakalisiwe ukuba alwa nemiphumo emibi ye-sickle hemoglobin (HbS) kwaye anciphise iimpawu ze-SCD kunye ne-thalassemia, kubandakanya ukuthintela iingxaki ze-vaso-occlusive kunye nokunciphisa i-hemolytic anemia.

I-BIOOCUS, iqela eliphambili kwicandelo lonyango lwezakhi zofuzo, ngokubambisana neSibhedlele iLu Daopei, ibe negalelo elikhulu ekuziseni olu nyango lutsha kwizigulana. 

Impumelelo yezonyango yolu nyango ayinakuthelekiswa nanto, kukho izigulane ezili-15 ezinyangiweyo ukuza kuthi ga ngoku, zonke zifikelele ekuphumleni ngokupheleleyo kunye nophuculo olubonakalayo kumgangatho wobomi bazo. Eli zinga lokuphiliswa eliyi-100% linyathelo elibalulekileyo ekulweni ne-SCD kunye ne-thalassemia.

Olu nyango luye lwaziwa kwihlabathi liphela, iingcali kwihlabathi liphela ziluncoma njengempumelelo ekunyangeni iingxaki zegazi ezibangelwa yimfuza. Luyahluka kungekuphela nje ngokusebenza kwalo kodwa nangokuxabisa kwalo. Ngokungafaniyo nezinye iindlela zonyango zemfuza, ezinokubiza kakhulu, olu nyango lubonelela ngemodeli yexabiso efikelelekayo, nto leyo eyenza ukuba lube lukhetho olusebenzayo kuluhlu olubanzi lwezigulane.

Kwityala elinye elinzima, isigulane esineminyaka eli-12 ubudala esineengxaki eziphindaphindayo ze-vaso-occlusive crisis kunye ne-hemolytic anemia enzima siye saphelelwa ziimpawu emva konyango ngolu nyango lwezakhi zofuzo. Eli tyala, phakathi kwezinye, ligxininisa amandla okuguqula unyango lwezigulane ze-SCD kunye ne-thalassemia kwihlabathi liphela.

Njengoko i-BIOOCUS iqhubeka nokwandisa ukufumaneka kolu nyango, kuquka novavanyo lwezonyango oluzayo eTshayina, ikamva libonakala lithembisa kwabo bachaphazeleka zezi zifo zinzima. Ngoncedo lweSibhedlele iLu Daopei, olu nyango luza kuchaza kwakhona umgangatho wokhathalelo lwe-SCD kunye ne-thalassemia, lubonelela ngesivumelwano esitsha sobomi kwizigulana ezingenakubalwa.

Ukuba wena okanye umntu omthandayo uchatshazelwa sisifo se-sickle cell okanye i-thalassemia kwaye unomdla wokuhlola olu nyango lutsha, siyakumema ukuba unxibelelane nathi. Iqela lethu likulungele ukukunika inkxaso nolwazi oludingayo ukuze wenze izigqibo ezinolwazi malunga nohambo lwakho lwezempilo.

Inkxaso epheleleyo kwizigulana zamazwe ngamazwe eziya eTshayina ukuze zifumane unyango lwe-Hemo-cell

Apha kwi-BIOOCUS, sizinikele ekuboneleleni ngononophelo olukhethekileyo kunye nenkxaso kwizigulane zamazwe ngamazwe ezine-thalassemia okanye isifo se-sickle cell ezifuna unyango lwe-Hemo-cell eTshayina. Iinkonzo zethu ezibanzi ziquka lonke inyathelo lohambo, ukusuka kumalungiselelo angaphambi kohambo ukuya kunyango lwasemva konyango.

1. Ukulungiselela Ngaphambi Kohambo

Ngaphambi kokuba bahambe, abaguli baya kuhlolwa ngokucokisekileyo kwizibhedlele zabo zasekuhlaleni. Oku kuquka uvavanyo olupheleleyo lwembali yezonyango, ukujonga iimpawu ezibalulekileyo, uvavanyo lwegazi, kunye nokuhlolwa okuthile okwenzelwe isifo se-thalassemia okanye i-sickle cell. Siqinisekisa ukuba onke amaxwebhu ezonyango, kuquka iirekhodi zotofelo-gazi kunye nokuqinisekiswa kokuchongwa, aqulunqwe ngokufanelekileyo kwaye athunyelwa kwiziko lethu ukuze lihlolwe.

2. Ukuhamba kunye nokulaliswa esibhedlele

Siyenza lula yonke inkqubo yokuhamba, kuquka nokulungiselela ukubhukisha izigulane ezilaliswe kwizibhedlele ezikhethiweyo. Izigulane zikhokelwa kwinkqubo yokwamkelwa xa zifika, ukuqinisekisa ukuba utshintsho olungenamthungo luhamba kakuhle kunyango.

3. Inkqubo yoNyango

Emva kokwamkelwa, izigulana ziya kuhlolwa uthotho lwangaphambi kokuxilongwa. Ukuba zithathwa njengezifanelekileyo, kuya kuqala ukuqokelelwa nokuhlanganiswa kweeseli ze-hematopoietic stem. Ezi seli zithunyelwa kwiziko lethu le-GMP ukuze kulawulwe umgangatho kwaye kwenziwe, apho izigulana zinokubuyela kwilizwe lazo ukuze zilinde eminye imiyalelo.

4. Ukufakelwa noNyango lokuLandela

Nje ukuba ii-stem cells ezilungisiweyo zivunyiwe, izigulane ziya kubuyela ukuze zifakelwe ezinye izigulana. Iqela lethu lizimisele ukubonelela ngenkxaso eqhubekayo kulo lonke ixesha lokulaliswa esibhedlele, ukuqinisekisa ukuba isigulane ngasinye sifumana unyango olukhethekileyo. Emva kokukhululwa, siququzelela uviwo lokulandelela rhoqo ukuze sijonge ukuchacha kunye nokuphila kakuhle.

E- I-BIOOCUS, sibeka phambili impilo nentuthuzelo yezigulane zethu zamazwe ngamazwe, senza konke okusemandleni ethu ukubonelela ngamava onyango axhasayo napheleleyo. Ukuze ufumane ulwazi oluthe kratya malunga neenkonzo zethu, nceda unxibelelane nathi.