Gene therapy: ndi chiyani, momwe zimachitikira komanso zomwe zitha kuchiritsidwa

Zamkati

• Momwe zimachitikira
• Njira ya CRISPR
• Njira Zamagalimoto A T-Cell
• Matenda omwe chithandizo cha majini chitha kuchiza
• Mankhwala a Gene motsutsana ndi khansa

Mankhwala a Gene, omwe amadziwikanso kuti geni kapena kusintha kwa majini, ndi njira yatsopano yopangira njira zomwe zitha kukhala zothandiza pochiza komanso kupewa matenda ovuta, monga matenda amtundu ndi khansa, posintha majini ena.

Chibadwa chimatha kutanthauziridwa ngati gawo lofunikira kwambiri kubadwa ndipo chimapangidwa ndi ma nucleic acid, ndiye kuti, DNA ndi RNA, komanso zomwe zimakhala ndi chidziwitso chokhudzana ndi mawonekedwe a munthuyo komanso thanzi lake. Chifukwa chake, chithandizo chamtunduwu chimakhala chomwe chimayambitsa kusintha kwa ma DNA am'magazi omwe akhudzidwa ndi matendawa ndikuyambitsa chitetezo cha mthupi kuti athe kuzindikira minofu yowonongeka ndikulimbikitsa kuthetsedwa.

Matenda omwe angachiritsidwe motere ndi omwe amakhudzana ndi kusintha kwa DNA, monga khansa, matenda amthupi, matenda ashuga, cystic fibrosis, mwa ena ofooka kapena matenda amtundu, komabe, nthawi zambiri amakhala ali mgulu la chitukuko mayesero.

Momwe zimachitikira

Mankhwala a Gene amaphatikizapo kugwiritsa ntchito majini m'malo mwa mankhwala ochizira matenda. Zimachitika posintha chibadwa cha minyewa yomwe imasokonezedwa ndi matendawa ndi yachilendo. Pakadali pano, mankhwala amtundu wachitidwa pogwiritsa ntchito ma molekyulu awiri, njira ya CRISPR ndi njira ya Car T-Cell:

Njira ya CRISPR

Njira ya CRISPR imakhala ndikusintha madera ena a DNA omwe atha kukhala okhudzana ndi matenda. Chifukwa chake, njirayi imalola kuti majini asinthidwe m'malo ena, mwanjira yeniyeni, yachangu komanso yotsika mtengo. Mwambiri, njirayi imatha kuchitidwa pang'ono:

• Ma jini apadera, omwe amathanso kutchedwa majini kapena chandamale, amadziwika;
• Pambuyo pozizindikiritsa, asayansi amapanga "chitsogozo cha RNA" chotsatira chomwe chimakwaniritsa dera lomwe akufuna;
• RNA iyi imayikidwa mchipindacho limodzi ndi puloteni ya Cas 9, yomwe imagwira ntchito podula momwe DNA ikuyendera;
• Kenako, DNA yatsopano imayikidwa muzochitika zapitazo.

Zosintha zambiri zamtundu zimakhudza majini omwe amapezeka m'maselo a somatic, ndiye kuti, maselo omwe ali ndi zinthu zomwe sizimafalikira kuchokera mibadwomibadwo, zomwe zimachepetsa kusintha kwa munthu ameneyo. Komabe, kafukufuku ndi zoyeserera zatulukira momwe njira ya CRISPR imachitikira pama cell a majeremusi, ndiye kuti, pa dzira kapena umuna, zomwe zatulutsa mafunso angapo okhudza kugwiritsa ntchito njirayi ndi chitetezo chake pakukula kwa munthuyo. .

Zotsatira zakanthawi yayitali za kusinthaku ndi kusinthika kwa majini sizikudziwikabe. Asayansi akukhulupirira kuti kusinthika kwa majini amunthu kumatha kupangitsa kuti munthu atengeke mosavuta pakusintha kwadzidzidzi, komwe kumatha kuyambitsa chitetezo chamthupi chambiri kapena matenda oyambira.

Kuphatikiza pa zokambirana zakusintha kwa majini kuthana ndi kuthekera kwakusintha kwadzidzidzi ndikusintha kwa kusinthaku m'mibadwo yamtsogolo, nkhani yamakhalidwe abwino idakambidwanso zambiri, popeza njirayi itha kugwiritsidwanso ntchito kusintha kwa mwana mawonekedwe, monga mtundu wa diso, kutalika, tsitsi, ndi zina zambiri.

Njira Zamagalimoto A T-Cell

Njira ya Car T-Cell imagwiritsidwa ntchito kale ku United States, Europe, China ndi Japan ndipo agwiritsidwa ntchito posachedwa ku Brazil kuchiza lymphoma. Njirayi imakhala yosinthira chitetezo cha mthupi kuti ma cell a chotupa azindikirike ndikuchotsedwa mthupi.

Pachifukwa ichi, maselo amtundu wa T amunthuyu amachotsedwa ndipo zinthu zawo zimapangidwa ndikuwonjezera mtundu wa CAR m'maselo, omwe amadziwika kuti chimeric antigen receptor. Pambuyo pakuwonjezera jini, kuchuluka kwa maselo kumakulitsidwa ndipo kuyambira pomwe ma cell amatsimikizika komanso kupezeka kwa zida zosinthira kuzindikira chotupa, kulowetsedwa kwa chitetezo chamthupi chamunthu, kenako, jakisoni ya maselo achitetezo osinthidwa ndi mtundu wa CAR.

Chifukwa chake, pali kutseguka kwa chitetezo cha mthupi, chomwe chimayamba kuzindikira ma cell a chotupa mosavuta ndipo chimatha kuthana ndi ma cellwa moyenera.

Matenda omwe chithandizo cha majini chitha kuchiza

Mankhwala a Gene akulonjeza kuti adzachiza matenda aliwonse amtundu, komabe, kwa ena okha omwe angathe kuchitika kale kapena ali mgawo loyesera. Kusintha kwachibadwa kwawerengedwa ndi cholinga chothandizira matenda amtundu, monga cystic fibrosis, congenital blindness, hemophilia ndi sickle cell anemia, mwachitsanzo, koma imawonekeranso ngati njira yomwe ingalimbikitse kupewa matenda owopsa komanso ovuta , monga mwachitsanzo khansa, matenda a mtima ndi kachilombo ka HIV, mwachitsanzo.

Ngakhale amaphunziridwa mozama pochiza ndi kupewa matenda, kusintha kwa majini kungagwiritsidwenso ntchito pazomera, kuti zizitha kulolera kusintha kwa nyengo komanso kulimbana ndi majeremusi ndi mankhwala ophera tizilombo, komanso muzakudya ndi cholinga chokhala ndi thanzi labwino .

Mankhwala a Gene motsutsana ndi khansa

Mankhwala a Gene a chithandizo cha khansa amachitika kale m'maiko ena ndipo amawonetsedwa makamaka pamatenda a leukemias, lymphomas, melanomas kapena sarcomas, mwachitsanzo. Chithandizo chamtunduwu chimakhala makamaka chokhazikitsa maselo oteteza thupi kuti azindikire zotupa ndikuzithetsa, zomwe zimachitika pobaya ma virus kapena ma virus m'thupi la wodwalayo.

Amakhulupirira kuti, mtsogolomu, chithandizo cha majini chidzagwira ntchito bwino ndikusintha njira zamankhwala zaposachedwa za khansa, komabe, popeza ikadali yotsika mtengo ndipo ikufuna ukadaulo wapamwamba, zikuwonetsedwa bwino ngati sizikugwirizana ndi mankhwala a chemotherapy, radiotherapy ndi opaleshoni.