Ukwelashwa Ngezakhi Zofuzo Okusha Kunikeza Ithemba Elisha Ngeziguli Ezinesifo Se-Sickle Cell Ne-Thalassemia

Ukwelashwa Ngezakhi Zofuzo Okusha Kunikeza Ithemba Elisha Ngeziguli Ezinesifo Se-Sickle Cell Ne-Thalassemia

Entuthukweni entsha yeziguli ezihlushwa yisifo se-sickle cell (SCD) kanye ne-thalassemia, ukwelashwa okusha kwezakhi zofuzo kubonisa impumelelo emangalisayo. Lokhu kwelashwa, okusebenzisa ubuchwepheshe obuthuthukisiwe bokuhlela izakhi zofuzo be-CRISPR/Cas9, kubonise izinga lokwelapha elingu-100% ezivivinyweni zemitholampilo, okunikeza ithemba elisha kulabo abalwa nalezi zinkinga ezinkulu zegazi.

Lolu hlobo lokwelapha, olwakhiwe ngeplatifomu ye-ModiHSC® eyimfihlo, luhlose izimpande zofuzo ze-SCD kanye ne-thalassemia. Ngokuhlela ngokunembile isithuthukisi se-BCL11A kumaseli e-hematopoietic stem kanye ne-progenitor, lolu hlobo lokwelapha luvumela umzimba ukuthi ukhiqize amazinga aphezulu e-hemoglobin yengane (HbF). Amazinga aphezulu e-HbF aboniswe ukuthi alwa nemiphumela elimazayo ye-sickle hemoglobin (HbS) futhi anciphise izimpawu ze-SCD kanye ne-thalassemia, okuhlanganisa nokuvimbela izinkinga ze-vaso-occlusive kanye nokunciphisa i-hemolytic anemia.

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

Impumelelo yezokwelapha yalolu hlobo lokwelapha ayinakuqhathaniswa, njengoba iziguli ezingu-15 zelashwe kuze kube manje, zonke zithola ukuphulukiswa okuphelele kanye nokuthuthuka okuphawulekayo ekhwalithini yazo yokuphila. Leli zinga lokuphulukiswa elingu-100% liyingqophamlando ebalulekile ekulweni ne-SCD kanye ne-thalassemia.

Ukwelashwa sekuqashelwe emhlabeni wonke, ochwepheshe emhlabeni wonke bekubonga njengokuphumelela ekwelapheni izifo zegazi ezibangelwa izakhi zofuzo. Kuvelele hhayi nje kuphela ngokusebenza kahle kodwa nangokusebenzisa kahle izindleko. Ngokungafani nezinye izindlela zokwelapha izakhi zofuzo, ezingabiza kakhulu, lokhu kwelashwa kunikeza imodeli yentengo efinyeleleka kalula, okwenza kube inketho efanelekile yeziguli eziningi.

Kwesinye isimo esicindezelayo, isiguli esineminyaka eyi-12 ubudala esinezinkinga eziphindaphindayo ze-vaso-occlusive kanye ne-hemolytic anemia enzima sabhekana nokuphela kwezimpawu ngokuphelele ngemuva kokwelashwa ngale ndlela yokwelapha izakhi zofuzo. Leli cala, phakathi kwezinye, liqokomisa amandla okuguqula ukwelashwa kweziguli ze-SCD kanye ne-thalassemia emhlabeni jikelele.

Njengoba i-BIOOCUS iqhubeka nokwandisa ukutholakala kwalolu hlobo lokwelapha, okuhlanganisa nezivivinyo zezokwelapha ezizayo eShayina, ikusasa libonakala lithembisa kulabo abathintekile yilezi zifo eziyinselele. Ngokusekelwa yiSibhedlela iLu Daopei, lolu hlobo lokwelapha luhlelelwe ukuchaza kabusha izinga lokunakekelwa kwe-SCD kanye ne-thalassemia, okunikeza ithuba elisha lokuphila kweziguli eziningi.

Uma wena noma othandekayo wakho nithintekile yisifo se-sickle cell noma i-thalassemia futhi ninesifiso sokuhlola lolu hlobo lokwelashwa olusha, sikumema ukuthi uxhumane nathi. Ithimba lethu likulungele ukukunikeza ukwesekwa nolwazi oludingayo ukuze wenze izinqumo ezinolwazi mayelana nohambo lwakho lokunakekelwa kwempilo.

Ukusekelwa Okuphelele Kweziguli Zamazwe Ngamazwe Eziya EShayina Ukuze Ziyokwelashwa Nge-Hemo-cell

Kwa-BIOOCUS, sizinikele ekuhlinzekeni ukunakekelwa okuvelele kanye nokusekelwa kweziguli zomhlaba wonke ezine-thalassemia noma isifo se-sickle cell ezifuna ukwelashwa kwe-Hemo-cell eShayina. Izinsizakalo zethu eziphelele zihlanganisa zonke izinyathelo zohambo, kusukela ekulungiseleleni ngaphambi kohambo kuya ekunakekelweni kwangemva kokwelashwa.

1. Ukulungiselela Ngaphambi Kohambo

Ngaphambi kokuhamba, iziguli zizohlolwa ngokucophelela ezibhedlela zasendaweni. Lokhu kuhlanganisa ukuhlolwa okuphelele komlando wezokwelapha, ukuqapha izimpawu ezibalulekile, ukuhlolwa kwegazi, kanye nokuhlolwa okuthile okwenzelwe isifo se-thalassemia noma i-sickle cell. Siyaqinisekisa ukuthi yonke imibhalo yezokwelapha, okuhlanganisa amarekhodi okumpontshelwa igazi kanye nokuqinisekiswa kokuxilongwa, ihlanganiswe kahle futhi idluliselwa esikhungweni sethu ukuze ibukezwe.

2. Ukuhamba Nokulaliswa Esibhedlela

Senza kube lula yonke inqubo yokuhamba, okuhlanganisa nokuhlela ukubhukha iziguli ezibhedlela ezikhethiwe. Iziguli ziqondiswa ngenqubo yokwamukelwa lapho zifika, okuqinisekisa ukuthi ukushintshela ekwelashweni kuhamba kahle.

3. Inqubo Yokwelapha

Ngemva kokwamukelwa, iziguli zizokwenza uchungechunge lokuhlolwa kokuqala. Uma kubhekwa njengezifanelekile, kuzoqala ukuqoqwa nokuhlanganiswa kwamaseli okuqala egazi. Lawa maseli athunyelwa esikhungweni sethu se-GMP ukuze kulawulwe ikhwalithi futhi kukhiqizwe, lapho iziguli zingabuyela khona emazweni azo ukuze zilinde imiyalelo eyengeziwe.

4. Ukufakelwa kanye Nokunakekelwa Kokulandelela

Uma amaseli okuqala alungisiwe esevunyiwe, iziguli zizobuya ukuze zifakelwe ezinye izitho. Ithimba lethu lizibophezele ekuhlinzekeni ukwesekwa okuqhubekayo phakathi nesikhathi sokulaliswa esibhedlela, ukuqinisekisa ukuthi isiguli ngasinye sithola ukunakekelwa komuntu siqu. Ngemva kokuphuma esibhedlela, sihlela ukuhlolwa okulandelanayo njalo ukuze siqaphe ukululama kanye nenhlalakahle.

Ku- I-BIOOCUS, sibeka impilo nenduduzo yeziguli zethu zomhlaba wonke phambili, senza yonke imizamo yokuhlinzeka ngolwazi lokwelapha olusekelayo noluphelele. Ukuze uthole ulwazi olwengeziwe mayelana nezinsizakalo zethu, sicela uxhumane nathi.