I-Innovative Gene Therapy Inikeza Ithemba Elisha Ngeziguli zeSickle Cell kanye ne-Thalassemia

I-Innovative Gene Therapy Inikeza Ithemba Elisha Ngeziguli zeSickle Cell kanye ne-Thalassemia

Entuthukweni edabukisayo yeziguli ezinesifo se-sickle cell (SCD) kanye ne-thalassemia, ukwelashwa okusha kwezakhi zofuzo kubonisa impumelelo emangalisayo. Lokhu kwelashwa, okusebenzisa ubuchwepheshe obuthuthukisiwe bokuhlela izakhi zofuzo ze-CRISPR/Cas9, kubonise izinga lokwelapha elingu-100% ezivivinyweni zomtholampilo, okunikeza ithemba elivuselelwe kulabo abalwa nalokhu kuphazamiseka kwegazi okunzima.

Ukwelashwa, okuthuthukiswe ngenkundla yokuphathelene ye-ModiHSC®, kuqondise izimpande zofuzo ze-SCD ne-thalassemia. Ngokuhlela kahle isithuthukisi se-BCL11A kusiqu se-autologous hematopoietic kanye namaseli azala, ukwelashwa kwenza umzimba ukwazi ukukhiqiza amazinga aphezulu e-fetus hemoglobin (HbF). Amazinga e-HbF aphakanyisiwe abonisiwe ukuthi amelene nemiphumela elimazayo ye-sickle hemoglobin (HbS) futhi anciphise izimpawu zakho kokubili i-SCD ne-thalassemia, okuhlanganisa nokuvinjelwa kwezinkinga ze-vaso-occlusive kanye nokunciphisa i-hemolytic anemia.

I-BIOOCUS, ibutho elihamba phambili emkhakheni wezokwelapha ngezakhi zofuzo, ngokubambisana neSibhedlela i-Lu Daopei, ibe nesandla ekuletheni lokhu kwelashwa okusha ezigulini. 

Impumelelo yokwelashwa emtholampilo ayinakuqhathaniswa, njengoba iziguli ezingu-15 zelashwa kuze kube manje, zonke zithole ukuthethelelwa okuphelele kanye nentuthuko enkulu eqophelweni lazo lempilo. Leli zinga lokwelapha elingu-100% liyingqopha-mlando ebalulekile ekulweni ne-SCD kanye ne-thalassemia.

Lokhu kwelashwa sekudume umhlaba wonke, kanti ochwepheshe emhlabeni wonke bekuncoma njengempumelelo ekwelapheni izifo zegazi eliwufuzo. Ayigqami nje kuphela ngokusebenza kwayo kahle kodwa futhi nangokubiza kwayo. Ngokungafani nezinye izindlela zokwelapha zofuzo, ezingabiza kakhulu, lokhu kwelashwa kunikeza imodeli yentengo efinyeleleka kakhudlwana, ikwenze kube inketho esebenzayo kuhlu olubanzi lweziguli.

Kwesinye isimo esiphoqayo, isiguli esineminyaka engu-12 ubudala esinezinkinga eziphindaphindayo ze-vaso-occlusive kanye ne-anemia enzima kakhulu ye-hemolytic sabhekana nokuqedwa okuphelele kwezimpawu ezilandela ukwelashwa ngalolu hlobo lokwelapha. Leli cala, phakathi kokunye, ligqamisa amandla okuguqula ukwelashwa kweziguli ze-SCD kanye ne-thalassemia emhlabeni wonke.

Njengoba i-BIOOCUS iqhubeka nokwandisa ukutholakala kwalokhu kwelashwa, okuhlanganisa nezivivinyo zomtholampilo ezizayo e-China, ikusasa libukeka lithembisa labo abathintwe yilezi zifo eziyinselele. Ngosizo lweSibhedlela i-Lu Daopei, lokhu kwelashwa kusethelwe ukuchaza kabusha izinga lokunakekelwa kwe-SCD ne-thalassemia, kunikeze impilo entsha ezigulini ezingenakubalwa.

Uma wena noma othandiweyo wakho nithintwa isifo se-sickle cell noma i-thalassemia futhi nithanda ukuhlola le mishanguzo yokwelapha, sikumema ukuthi usithinte. Ithimba lethu likulungele ukukunikeza ukwesekwa nolwazi oludingayo ukuze wenze izinqumo ezinolwazi mayelana nohambo lwakho lokunakekelwa kwezempilo.