Zvitsva zvakawanikwa kubva kuItaly kurwisa Hurler syndrome
Zvitsva zvakakosha zvekurapa zvakawanikwa kurwisa Hurler syndrome
Chii chinonzi Hurler syndrome
Chimwe chezvirwere zvisingawanzoitiki zvinogona kuitika muvana Hurler chirwere, inozivikanwawo se“mukopolysaccharidosis mhando 1H“. Ichi chirwere chisingawanzo chinokanganisa Mwana 1 pa100,000 XNUMX voga voga kuberekwa patsva. Zvinosanganisira kushaikwa kweimwe enzyme inokonzera kukanganisa shuga chaiyo, glycosaminoglycans. Kuunganidzwa kweshuga idzi kunokonzera kukuvadzwa kwemasero, kukanganisa kukura uye psycho-cognitive kukura kwevana.
Zvinosuruvarisa, mhedzisiro yacho yakaipa, uye rufu runogona kuitika kubva pakutanga kwekuyaruka, kunyanya nekuda kwemwoyo kana matambudziko ekufema.
Nzvimbo itsva yezvokurapa
Yatova muna 2021, tsvagiridzo kubva ku San Raffaele Telethon Institute yeGene Therapy yanga yaratidza mhedzisiro inovimbisa. Iyi tsika inosanganisira kupa yakagadziridzwa vhezheni yeruzivo rwemajini runodiwa kuburitsa iyo enzyme inorasika.
Hunhu hwekurapa huri mukushandisa, mukugadzirisa kwemurwere hematopoietic stem masero, s.ome vectors anobva kuHIV, utachiona hunokonzera AIDS. Zvinofanira kucherechedzwa kuti kuwedzera, zvikamu zvidiki zvekutevedzana kwepakutanga zviri kushandiswa mumunda wejini kurapwa kwezvirwere zvisingawanzo.
Chidzidzo chitsva chakaburitswa mujenari JCI Insight, yakaitwa nevaongorori vepasi rese vachitungamirwa neSapienza University yeRome uye Tettamanti Foundation yeMonza, nemipiro kubva kuIrccs San Gerardo dei Tintori Foundation yeMonza uye University of Milano-Bicocca, yakabvumira kugadzirwa kwerabhoritari. organoid yemapfupa, shanduro yakapfava uye ine mativi matatu enyama inoumba mapfupa uye shwashwa mumuviri womunhu.
Izvi zvichapera chiedza chitsva paHurler syndrome.
Kubvunzurudzwa naAnsa, Vanachiremba Serafini uye Riminucci, vanyori vanyori vechidzidzo naSamantha Donsante weSapienza naAlice Pievani weTettamanti Foundation sevasina kutungamira, vakataura kuti kusikwa kweiyi organoid hakuzovhurike chete. mikova mitsva yekugadzirisa Hurler syndrome asiwo kudzamisa tsvakiridzo yakanangana neiyo kurapwa kwezvimwe zvirwere zvakakomba zvemajini.
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