Isifo seSickle Cell kunye neThalassemia

KwiZiko loNyango lwaMazwe ngaMazwe leBIOOCUS, siyazingca ngokwazisa I-Hemo-cell, unyango lwezakhi zofuzo oluyintshayelelo olunika ithemba elitsha kwizigulana ezineengxaki isifo se-sickle cell (SCD) kwaye i-thalassemia (TDT) exhomekeke ekutofelweni kwegaziIHemo-cel yenzelwe ukujongana nonobangela oyintloko wezi meko, inika unyango olunokwenzeka ngonyango oluqhutywa kube kanye.

Yintoni iHemo-cell?

I-Hemo-cel lunyango oluphambili olusebenzisa iiseli ze-hematopoietic stem kunye ne-progenitor zesigulana (ii-HSPC), eziguqulwa ngokwemfuza ukuze zivelise i-hemoglobin esempilweni. Ngokulungisa i-gene engalunganga ebangela ukuphazamiseka kwegazi, i-Hemo-cel inokuphucula kakhulu ubomi bezigulana, inokuphelisa isidingo sotofelo-gazi rhoqo kwaye inike isisombululo sobomi bonke.

Isebenza njani iHemo-cell?

Inkqubo yonyango iqala ngokuqokelela ii-HSPC egazini lesigulana. Ezi seli emva koko ziyaphuculwa ngaphandle komzimba kusetyenziswa i-lentiviral vector engenisa i-β-globin gene eguquliweyo. Nje ukuba iiseli zilungele, zibuyiselwa kwisigulana, apho ziqala khona ukuvelisa iiseli ezibomvu zegazi eziphilileyo ezikwaziyo ukuvelisa i-hemoglobin esebenzayo. Le nkqubo ibuyisela ibhalansi phakathi kwe-α-globin kunye ne-β-globin chains egazini, inciphisa ngokufanelekileyo iimpawu ze-anemia kwaye ivumela isigulana ukuba sifezekise ukuzimela ekutofeleni igazi.

Iziphumo eziQinisekisiweyo

Uvavanyo lwezonyango lweHemo-cell lubonisile impumelelo emangalisayo:

• I-100% yezigulane kwilingo baphumelele ekufakweni komongo wethambo.
• Uninzi lwezigulane lwaba ngaphandle kotofelo-gazi kwiiveki ezimbalwa emva konyango.
• Omnye umguli ebengenalo utofelo-gazi phantse kangangexesha elide iminyaka emine ngaphandle kwemiphumo emibi ebalulekileyo.
• Olu nyango lukwanceda ekunciphiseni ukugcwala kwesinyithi, ingxaki eqhelekileyo kwizigulana ze-TDT ezitofelwa igazi rhoqo.

Enye indlela ekhuselekileyo

Ngokungafaniyo nonyango lwendabuko olufana nokufakelwa kweeseli ze-hematopoietic stem cell (HSCT), olufuna umntu oza kuxhasa kwaye luthwala iingozi ezifana nesifo se-graft-versus-host (GVHD), I-Hemo-cell isebenzisa iiseli zesigulana, ukuphepha ezi ngxaki. Oku kwenza unyango ikhuselekile kwaye ifikeleleka lula kwiintlobo ngeentlobo zezigulana, ngaphandle kwesidingo sonyango oluhlala ixesha elide lokunciphisa umngcipheko wokugula.

Ulwando Lwethu Lwehlabathi

Ekubeni siphumelele kakhulu eTshayina, ngoku sandisa ukufumaneka kweHemo-cel kwihlabathi liphela. Ngokubambisana kwezonyango e-China eSaudi Arabia, eThailand, eMalaysia, nangaphezulu, sizimisele ukwenza olu nyango lutsha lufikeleleke kwizigulana kwihlabathi liphela.

Ikamva Elingenalo Utofelo-gazi

I-Hemo-cel inika ikamva elithembisayo kwabo bachaphazeleka yi-SCD kunye ne-beta-thalassemia. Khawuthelekelele ubomi obungenalo utofelo-gazi oluqhubekayo, olunonyango oluhlala luhleli. Apha kwi-BIOOCUS, sizimisele ukwenza lo mbono ube yinyani ngezisombululo zonyango lwezakhi zofuzo eziphambili ezifana ne-Hemo-cel.

Qhagamshelana nathi namhlanje ukuze ufunde ngakumbi ngendlela iHemo-cel enokutshintsha ngayo uhambo lwakho lonyango kwaye inike ikamva eliqaqambileyo nelinempilo.