Hāʻawi ʻo Innovative Gene Therapy i ka manaʻolana hou no nā maʻi Sickle Cell a me Thalassemia

Hāʻawi ʻo Innovative Gene Therapy i ka manaʻolana hou no nā maʻi Sickle Cell a me Thalassemia

Ma kahi hoʻomohala hoʻomohala honua no nā poʻe maʻi e loaʻa ana i ka maʻi sickle cell (SCD) a me ka thalassemia, ke hōʻike nei kahi lāʻau lapaʻau hou i ka kūleʻa kupaianaha. ʻO kēia lāʻau lapaʻau, e hoʻohana ana i ka ʻenehana hoʻoponopono gene CRISPR/Cas9, ua hōʻike i kahi 100% lāʻau lapaʻau i nā hoʻokolohua lapaʻau, e hāʻawi ana i ka manaʻolana hou i ka poʻe e hakakā nei i kēia mau maʻi koko koʻikoʻi.

ʻO ka lāʻau lapaʻau, i hoʻomohala ʻia me ka platform ModiHSC® proprietary, kuhikuhi i nā aʻa genetic o SCD a me thalassemia. Ma ka hoʻoponopono pololei ʻana i ka mea hoʻonui BCL11A i loko o ka autologous hematopoietic stem a me nā cell progenitor, hiki i ka lāʻau lapaʻau ke hiki i ke kino ke hana i nā pae kiʻekiʻe o ka hemoglobin fetal (HbF). Ua hōʻike ʻia nā pae kiʻekiʻe o ka HbF e pale aku i nā hopena pōʻino o ka sickle hemoglobin (HbS) a hōʻemi i nā hōʻailona o ka SCD a me ka thalassemia, me ka pale ʻana i nā pilikia vaso-occlusive a me ka hoʻēmi ʻana i ka anemia hemolytic.

ʻO BIOOCUS, he alakaʻi alakaʻi i ke kahua o ka lāʻau lapaʻau gene, me ka hui pū ʻana me ka Halemai Lu Daopei, i mea kōkua i ka lawe ʻana i kēia lapaʻau hou i nā maʻi. 

ʻAʻole kūlike ka holomua o ka lāʻau lapaʻau, me 15 mau mea maʻi i mālama ʻia a hiki i kēia manawa, loaʻa nā mea āpau i ke kala piha a me nā hoʻomaikaʻi nui i ko lākou ola. ʻO kēia 100% lāʻau lapaʻau he mea nui i ka hakakā ʻana me SCD a me thalassemia.

Ua loaʻa i ka lāʻau lapaʻau ka ʻike honua, me nā poʻe loea ma ka honua holoʻokoʻa e haʻi nei iā ia ma ke ʻano he holomua i ka mālama ʻana i nā maʻi koko genetic. ʻAʻole wale ia no ka maikaʻi akā no ka maikaʻi hoʻi. ʻAʻole like me nā lāʻau lapaʻau ʻē aʻe, hiki ke pāpā ʻia, hāʻawi kēia lapaʻau i kahi kumu kumu kūʻai maʻalahi, e lilo ia i koho kūpono no kahi ākea ākea o nā maʻi.

I kekahi hihia koʻikoʻi, ua ʻike ka mea maʻi he 12 makahiki me nā crises vaso-occlusive mau a me ka anemia hemolytic koʻikoʻi i ka pau ʻana o nā hōʻailona ma hope o ka mālama ʻana me kēia ʻano lāʻau lapaʻau. ʻO kēia hihia, i waena o nā mea ʻē aʻe, e hōʻike ana i ka hiki ke hoʻololi i ka lāʻau lapaʻau no nā maʻi SCD a me thalassemia ma ka honua holoʻokoʻa.

Ke hoʻomau nei ʻo BIOOCUS i ka loaʻa ʻana o kēia lāʻau lapaʻau, me nā hoʻokolohua lapaʻau e hiki mai ana ma Kina, ʻike ʻia ka wā e hiki mai ana no ka poʻe i loaʻa i kēia mau maʻi paʻakikī. Me ke kākoʻo o ka Halemai Lu Daopei, ua hoʻonohonoho ʻia kēia lāʻau lapaʻau e wehewehe hou i ke kūlana o ka mālama ʻana no SCD a me thalassemia, e hāʻawi ana i kahi hoʻolimalima hou i ke ola no nā maʻi he nui.

Inā pilikia ʻoe a i ʻole kekahi mea aloha i ka maʻi sickle cell a i ʻole thalassemia a makemake ʻoe e ʻimi i kēia lāʻau lapaʻau hou, kono mākou iā ʻoe e kelepona iā mākou. Mākaukau kā mākou hui e hāʻawi iā ʻoe i ke kākoʻo a me ka ʻike e pono ai ʻoe e hana i nā hoʻoholo ʻike e pili ana i kāu huakaʻi mālama ola.