Tariro itsva kune vanhu vane thalassemia, ndeupi mushonga mutsva?

Imwe nyanzvi munyaya dzekurapa mukurapa thalassemia inoti 2018 inogona kushandura marapirwo echirwere ichi chinopedza simba chinokanganisa ropa. Dr. Rabih Hanna, chiremba wezvirwere zveropa muvana, oncologist uye mazvikokota wekutamisa mwongo paCleveland Clinic kuU.S., akati nhevedzano yebudiriro yezvokurapa ichangobva kuitika mundima ye<em>genetic therapy inoreva kuti kurapa thalassemia “kunenge kwava pedyo.”

Chirevo chaDr. Hanna chakauya pachiitiko cheInternational Thalassemia Day, iyo inowira musi wechisere waMay wegore rega rega, iyo yaakasimbisa, zvichibva pane ichangobva kudhindwa data rekutsvaga, kuti kurapwa "kunogona kunge kwave kuripo", tichifunga kuti genetic. kurapwa kwave nemakore kwakaratidza mukana wekugadzira kugona kurapa kubva kuIyi genetic disorder, kunyanya nekubudirira kwekusvetuka kukuru mundima dzesainzi yekutanga, uye akawedzera kuti: "Tinoona kuti mapurojekiti ekutanga ekutsvagisa mundima iyi ari kushanduka kuita nzira dzekurapa, uye mumwedzi mishoma yapfuura takaona tsvakiridzo yakanaka yakaburitswa kubva muongororo muzvidzidzo zvevanhu, uko The gene therapy inoshanda, mhedzisiro ndeyechigarire, uye varwere havana chero mhedzisiro inotyisa. " Dr. Hanna akataura tariro yake yokuti “tiri kutarisira zvomenemene kufambira mberi negadziriro yokurapa inobudirira mune ramangwana rinooneka.”

Thalassemia chirwere chemajini chinokonzera kusagadzikana muhemoglobin muropa, inova chikamu chemasero matsvuku eropa ane basa rekufambisa okisijeni. Migumisiro yaro inobva kuanemia, iyo inokonzera kuneta uye ganda rakacheneruka, kumatambudziko emapfupa uye spleen yakakura. Ongororo yehuremerwa hweupfumi hwenyika yose hwethalassemia hunoti kune vanhu vanosvika 280 miriyoni vane chirwere ichi pasi rose, kusanganisira vanhu vanosvika 439,000 16,800 vari kurwara nechirwere chethalassemia, uye chirwere ichi chakakonzera kufa kwevarwere 2015 XNUMX muna XNUMX.

Thalassemia inowanikwa munharaunda dzinogara kuMiddle East, Mediterranean, South Asia neAfrica, uye inowanzobatanidzwa mukuongororwa kwemajini vasati varoorana mune dzimwe nzanga idzi.Mushonga unonyanyozivikanwa wevarwere vakanyanya kuisirwa ropa nguva dzose kuti ubvise zviratidzo. iyo inogara kwenguva refu.hupenyu uye ine maside effects. Nepo chete kurapwa kuripo parizvino kwethalassemia kuisirwa mwongo, iyo inogona kuitwa pachikamu chidiki chevarwere.

Muchirevo chechinyorwa chino, genetic therapy inovhura musuwo wemikana mitsva yekurapa nekurapa chirwere, nekushandura zvikamu zveiyo genetic code inokonzeresa. Varwere veThalassemia, mune imwe yezviyedzo zvekurapa zviri kuitika munzvimbo nhanhatu pasirese, vaive nemasero madiki akabudiswa kubva mumwongo wemapfupa avo.Mwongo wevarwere kubva kuzvirwere zvine chirwere kuburikidza nechemotherapy, maseru akagadziridzwa asati apinzwa mumongo. ropa kuti vawane nzira yokudzokera kumwongo, kwavakakura ndokuva masero matsvuku eropa anobudisa hemoglobin ine utano.

Migumisiro yacho, yakabudiswa muNew England Journal of Medicine, yakaratidza kuti kurapwa kwacho kwakaderedza zvikuru nhamba yokuisirwa ropa kwaidiwa nomurwere mumwe nomumwe. Varwere vakanga vaongororwa kwenguva iyo April apfuura yakasvika mwedzi 42 kubvira pakatanga kurapwa kwemajini, sezvo nhamba yokuisirwa ropa panguva iyoyo yakaderera nokusvika 74 muzana muzvirwere zvakakomba, nepo varwere vakawanda vaiva nemhosva dzisina kunyanya kuoma vakaita. kusadzoka Vanoda kuisirwa ropa.

Dr. Hanna akasimbisa kuti izvi zviri kuitika mune zvokurapa zvichatora nguva kuti zviwanikwe sekurapa, asi akasimbisa kuti zviri kuitika zviri panguva apo “kukunda mukutsvakurudza kunochinja kuva kukunda mukurapa,” tichifunga kuti izvi “zvinopa tariro yechokwadi kuvarwere vakawanda. vanorwara nethalassemia nhasi.” .

Nekune rimwe divi, imwe tekinoroji inovimbisa yave kufamba mumiedzo yevanhu ndeye CRISPR, kana kuti inogara yakapatsanurwa inodzokorodza cluster, chishandiso chinogona kushandura kutevedzana kweDNA uye kushandura basa remajini. Zvichienderana nemaitiro ekudzivirira anowanikwa mumabhakitiriya, CRISPR chishandiso chinozvipinza mukati memukati mejini uye chinocheka chikamu chinokanganisa cheDNA, pane kutsiva chine hutano neane chirwere.

Kambani yebiotechnology CRISPR Therapeutics nguva pfupi yadarika yakawana mvumo kubva kuzviremera zvakakodzera muEurope yekushandisa tekinoroji yekugadzira majini mukuyedza kurapa thalassemia, umo maseru anozogadziridzwa murabhoritari uye majini akagadziridzwa anodzoserwa mumuviri kuburikidza nekuwedzerwa ropa. Chimwe chirongwa chakasiyana paStanford University chiri kuronga kuyedza kurapa chirwere chegomarara, chirwere chemajini chinogovana zvakawanda zvakafanana nethalassemia, umo magadzirirwo emajini anoitwa mukati memuviri.

Dr. Hanna akasimbisa kuti kurapa kukuru kuripo iye zvino, kuisira ropa, “chinongova chigadzikiso chenguva pfupi, zvikurukuru kuvanhu vane hosha yakakomba,” achisimbisa kuti kuisirwa ropa pachakwo “kunotungamirira nokufamba kwenguva kuzvinetso zvoutano, tichifunga kuti kuwanda kwesimbi muropa kunotungamira Kuitika kwechirwere chemoyo nechiropa, hutachiona uye osteoporosis, izvo zvinoita kuti mhedzisiro iyi irebe nguva yekurapa. " Akagumisa kuti kubudirira mukuderedza kuisirwa ropa kunodiwa kuchengetedza nhamba yakakodzera yemasero matsvuku eropa “mugumisiro wakanaka kumurwere,” uye akawedzera, kuti: “Pfungwa yokuti kune chiverengero chakati chokurapa kutsva chiri pedyo, kwete kurapa kutsva. Kurapwa kumwe chete, kunoita kuti tive netariro yekuwana dzimwe nzira dzekurapa dzisingabatsiri.