Momwe mankhwala a EB amagwirira ntchito

Gene therapy ndi njira yochizira a chibadwa mwa kukonza zolakwika mu chibadwa cha munthu kuti ali ndi udindo zizindikiro zawo. Izi ndizosiyana pochiza zizindikiro za munthu payekha. Majini ndi maphikidwe a mapuloteni omwe matupi athu ali zopangidwa ndi. Akhoza kukhala ndi zolakwika mwa iwo zomwe zimalepheretsa mapuloteni ogwira ntchito kuti asapangidwe. Pamene thupi la munthu sangathe kupanga mapuloteni ena apakhungu, izi zimayambitsa zizindikiro za EB. 

Kuchiza kwa ma gene kumagwiritsa ntchito njira zachilengedwe kuchokera ku ma virus ndi mabakiteriya kupanga majini ogwira ntchito ndikuyika m'maselo momwe jini ikusowa kapena kusweka. Izi zikhoza kuchitika mwina potengera chitsanzo cha maselo a munthu mu labotale kuti akonze chibadwa chake kenako n’kuwabweza (amenewa amatchedwa ex vivo) kapena kumuthandiza munthu mwachindunji ndi jekeseni kapena gel osakaniza amene amaika jini yogwira ntchito m’maselo. m’thupi mwawo amene amachifuna (izi zimatchedwa mu vivo).  

Kulowetsa majini atsopano m'maselo athu kungakhale kovuta koma jini yatsopano, yolondola itayikidwa m'maselo a munthu imatha kugwiritsidwa ntchito ndi selo kuti iyambe kupanga mapuloteni omwe analibe. 

Ma virus nthawi zambiri amagwiritsidwa ntchito kuyika majini atsopano m'maselo chifukwa izi ndi zomwe amachita mwachibadwa. Kuchiza kwa ma gene kumapangitsa kachilombo kukhala kosavulaza pochotsa kuthekera kwake kodzibwereza yokha. Zimapangitsa kachilomboka kukhala kothandiza posintha jini yomwe ikanayika m'maselo athu kuti tidwale ndi jini yatsopano yomwe ingatipangitse kukhala athanzi. Njira ina yopezera majini m'maselo athu ndi kuwapaka ndi mafuta kapena mapuloteni. Majini paokha amawonongeka mosavuta ndi kuwala kwa dzuwa komanso mapuloteni obwera mwachilengedwe otchedwa ma enzyme. 

Vidiyo iyi yochokera ku American Society of Gene and Cell Therapy ikufotokoza za chithandizo cha majini:  

Ma virus osiyanasiyana amagwiritsidwa ntchito kuyika majini akulu kapena ang'onoang'ono (maphikidwe a protein aatali kapena afupi) m'maselo athu. 

Ma virus amenewa asinthidwa kotero kuti sangathe kutidwalitsa, koma mwina tinali ndi matenda obwera chifukwa cha mtundu wa ma virus omwe amagwiritsidwa ntchito popanga gene. Izi zikutanthauza kuti titha kukhala ndi chitetezo chamthupi ku mu vivo therapy pogwiritsa ntchito mtundu wa virus. Titha kukhalanso ndi chitetezo chamthupi ngati mu vivo gene therapy ibwerezedwa. Chithandizo cha majini sichingagwire ntchito bwino m'matupi athu ngati chitetezo chathu cha mthupi chimawononga kachilombo ka gene therapy isanapereke jini yatsopanoyo m'maselo athu.  

Nthawi zina chitetezo chathu cha mthupi chimakhudzidwa ndi mapuloteni atsopano, olondola ngati kuti ndi majeremusi. Ofufuza ayenera kufufuza kuti chithandizo cha majini sichimayambitsa kuyankha kwa chitetezo cha mthupi. 

Mitundu ina ya chithandizo cha majini imafuna kukhala chithandizo chamankhwala amodzi pomwe ina imafunikira kubwereza mobwerezabwereza. 

Khungu limakonzedwanso mosalekeza; maselo akale a khungu amafa ndikutuluka ndipo maselo atsopano amawalowa m'malo. Maselo atsopanowa amachokera mkati mwa khungu momwe maselo amakulira, kupanga kopi yatsopano ya majini awo onse ndikugawaniza awiri mobwerezabwereza kuti apange maselo atsopano a khungu. 

Maselo omwe ali ndi majini atsopano oikidwa mwa iwo ndi gene therapy amafa mwachibadwa ndi kusinthidwa ndi maselo atsopano kotero kuti chithandizo cha majini chingafunikire kubwerezedwa. Jini yatsopanoyo ingokoperedwa m’maselo atsopano ngati yakhala mbali ya imodzi mwa ma chromosome athu. Izi zimatchedwa kuphatikiza.  

Ma chromosome ndi zidutswa zazitali za DNA zokwiriridwa mkati mwa maselo athu ndipo jini iliyonse ndi gawo la imodzi mwa ma chromosomes. Ngati tiganiza za jini iliyonse ngati "maphikidwe" opangira mapuloteni omwe matupi athu amapangidwa kuchokera, chromosome iliyonse imakhala ngati bukhu la maphikidwe. Kuyika Chinsinsi chatsopano m'modzi mwamabuku opangira maphikidwe kumatanthauza kuti akopedwa. 

Njira zina zochiritsira za majini zidzaphatikiza jini yatsopano mu chromosome, ndipo ena sadzatero. Izi zitha kusintha utali wa mankhwalawo kuti athetse zizindikiro. 

Ngati jini yatsopano ikuphatikizidwa, ndikofunikira kuti isasokoneze jini ina iliyonse ndikuyimitsa kugwira ntchito. Tangoganizani kuti mwayimitsa Chinsinsi chatsopanocho mwangozi pagawo lina. Ochita kafukufuku ayenera kuonetsetsa kuti chithandizo cha majini sichidzayambitsa zolakwika m'majini ena aliwonse omwe alipo. 

Kusintha kwa ma gene ndi mtundu wa chithandizo cha jini chomwe chimachokera ku njira zachilengedwe zomwe zimagwiritsidwa ntchito ndi mabakiteriya kuti adziteteze ku ma virus. Mukhoza kuwerenga zambiri za izo Pano.

M'malo mopereka njira yatsopano, yogwiritsira ntchito majini ku maselo kuti athe kupanga mapuloteni osowa, kusintha kwa majini ndi njira yokonzekera jini yomwe ilipo, yosweka kuti akonze njira ya munthu. 

Ndikofunikira kuti ndondomekoyi isasinthe kwina kulikonse komwe kungayambitse zolakwika mu majini ena. Ndikofunikiranso kuti palibe kusintha komwe kumapangidwa ku dzira kapena ma cell a umuna zomwe zingatanthauze kuti mwana wabadwa ndi kusintha kwa majini komwe sanavomereze.  

Kusintha kwa ma gene kumachitika ngati mankhwala amtundu wa ex vivo m'malo mwa mankhwala a vivo. Maselo amtundu wa munthu amatha kusonkhanitsidwa, kuwongolera zolakwika zamtundu wake, ndikubwezeredwa kwa iwo. 

Kanemayu akufotokoza za mtundu wakusintha kwa majini pogwiritsa ntchito CRISPR/Cas9 system: 

Mankhwala ena omwe angakhalepo a EB amachokera ku maselo amtundu, mtundu wina wa selo lomwe nthawi zambiri limatengedwa kuchokera m'mafupa koma likhoza kubwera kuchokera ku ziwalo zina za thupi la wopereka, zomwe zingasinthe kukhala mitundu ina ya selo. Kuchiza kwa ma cell kumatha kuyika ma stem cell a munthu yemwe alibe EB kulowa m'magazi a munthu yemwe ali ndi EB. Maselowa amatha kupita ku khungu ndi ziwalo zina za thupi zomwe zimakhudzidwa ndi EB ndikukhala maselo omwe amatha kupanga mapuloteni osowa omwe amachititsa zizindikiro za EB. Izi zitha kukhala njira yochizira zizindikiro za EB kuzungulira thupi lonse. 

Maselo a Mesenchymal stromal (MSCs) ndi mtundu wa selo lomwe lili ndi zinthu zofanana ndi maselo oyambira omwe akuyesedwa mu EB. 

Dziwani zambiri za ma stem cell muvidiyoyi: 

Thandizo la mankhwala ndi pamene zizindikiro zimachiritsidwa ndi zinthu zomwe zimakhudza momwe matupi athu amagwirira ntchito. Zitha kukhala mapiritsi omwe timameza, jekeseni pakhungu kapena minofu, kuikidwa magazi kudzera mu singano mumtsinje wamagazi kapena zonona, kupopera, gel kapena dontho la diso. 

EB ndi matenda otupa, ndipo madokotala amamvetsetsa bwino momwe kutupa kumachitikira m'matupi athu. Izi zikutanthauza kuti amatha kusankha mankhwala omwe amachepetsa kutupa muzinthu zina kuti kubwereza za EB. 

Kupweteka kwa mabala a EB kumatha kuchiritsidwa ndi mankhwala ochepetsa ululu kapena mankhwala ophatikizira. 

Kanemayu akufotokoza momwe mankhwala angagwire ntchito m'matupi athu: 

Thandizo la puloteni limaphatikizapo kusintha puloteni yomwe ikusowa mwa anthu omwe ali ndi EB chifukwa cha kusintha kwa majini mu njira yolembera.  

M'malo mosintha njira yopangira puloteni yapakhungu (mankhwala othandizira), Thandizo la puloteni limayesa kubwezeretsa "chosakaniza" cha puloteni chomwe chikusowa pakhungu chomwe sichikuyenda bwino. Zili ngati kuyesa kuwonjezera kudzaza mu chitumbuwa chanu mutatuluka mu uvuni m'malo mosintha maphikidwe a pie osweka. Ochita kafukufuku ayenera kumvetsetsa momwe angafikitsire puloteni kumalo komwe ikuyenera kukhala. Izi zitha kukhala ndi jakisoni, kudzera m'magazi omwe amanyamula thupi lonse kapena kupita ku maso kapena zilonda zomwe khungu silimatchinga. 

Mapuloteni ofunikira pakuchiritsa kwa mapuloteni amatha kupangidwa m'malo a labotale momwe mabakiteriya kapena maselo a yisiti omwe ali ndi njira yolondola ya chibadwa amakula ndikugwiritsidwa ntchito ngati mafakitale ang'onoang'ono a mapuloteni. Tekinoloje iyi imatha kupanga bwino mapuloteni ambiri amunthu. Mwachitsanzo, insulin yaumunthu imapangidwa mwanjira imeneyi kuti ithandizire anthu odwala matenda ashuga.