kia ora therapy ira New tamarikitanga huru ārai

New gene therapy may cure childhood immune condition

 

Powered e Guardian.co.ukTēnei tuhinga ui “kia ora therapy ira New tamarikitanga huru ārai” e Hana, Devlin i tuhituhia, kaituhi pūtaiao, hoki theguardian.com i runga i Rātū 21 Paengawhāwhā 2015 17.01 UTC

puta tamariki ki te huru ārai kino ki te kua ora, i muri i te riroraa ei te tuatahi ki te kia hoatu he puka hou o te therapy ira.

Te kore te maimoatanga, nga tūroro e mamae i te huru onge huaina Wiskott-Aldrich syndrome, fehangahangai matapae oranga poto, me te wā roa i roto i te hōhipera.

Doctors i Great Ormond Street Hospital, nana nei i arahi te tamataraa angitu, ka mea nga kitenga tohua he wāhi koki mō te therapy ira a taea apu te ara mō ngā tūāhua rite te whakamahia hoki ngā āhuatanga ake noa, tae atu te mate pūtau toronaihi, me te thalassemia.

Ahorangi Adrian Thrasher, te immunologist nei arahina te mahi, mea: "Ko te tauira te tino kaha o te pehea ira therapy taea whakahere maimoatanga tino whai hua mō te tūroro ki te mate ira matatini me te taumaha tenei. Te reira i hoki excitingly whakaatu te pūmanawa mo te maimoatanga o te maha nui o ētahi atu mate mo e he rānei ta'efakafiemālie wātea ranei whakaora ngā. "

Nga kitenga whai rave rahi atu hua tamataraa pai i roto i te tau mua, e mea e aivanaa maka ana ki te okioki pariraa e kua te kupu whakaari o te therapy ira kua runga-hyped.

Emma Morris, he immunologist i University College London nei te ārahi i tēnei wā i te tamataraa ira therapy mō kanisā, mea: "Kua People mahi i runga i taua mea mo te kotahi tekau nga tau, me te ngā haere i teie nei te reira i te mooni e humarie taea e koe ira whakarerekē pūtau me te whakamōhio ia ratou ki te iwi."

I roto i te ako hou, e whitu nga tamariki ki Wiskott-Aldrich syndrome (KO) i tukinotia i Great Ormond Street Hospital me te whare hauora motuhake i roto i Paris.

pānga te huru ira i waenganui i tetahi, me te 10 tamariki i roto i nga miriona te ao me te whakaiti ratou kaha ki te whawhai mate. Tohumate taea ngā wāhanga toto, katoatia me ētahi atu mate kiri recurrent, me te mate autoimmune, a i roto i ngā take kino taea tamariki Me ki te noho wā roa i roto i te hōhipera.

Ka taea te tukinotia pai te huru ki te huaranga wheua wheua i te kaituku hauora, engari whakawhirinaki tenei i runga i te kimi i te ōrite pai mo te manawanui. "Ki te kore koe e whai i te kēmu pai, Ko uaua te maimoatanga me te titau rota o raau taero,"Ka mea a Thrasher.

nga tamariki katoa i roto i te tamataraa i mate kiri recurrent, te nuinga o te pakaruhanga toto mātanga me tetahi o nga tamariki i herea ki te wīra.

Hoki te maimoatanga hou, ka mau te pūtaiao ake wheua wheua o nga tamariki, me te kaiao te reira te whakamahi i te huaketo te kinokore ki te whakauru i te putanga mahi o te ira hē ki ngā pūtau ārai o te manawanui.

i hoatu ano e nga tamariki hahau, e whakangaro etahi o ngā wheua wheua o te tinana, i mua i whakakapia te pūtau kaiao.

E whai ake nei i te maimoatanga, i mate tetahi o nga tamariki, i raruraru i mua-ngā auauhia ki te maimoatanga. whakakitea pūnaha ārai nga atu e ono nga tama a te ora faahiahia, me te nuinga o ratou tohu whakatauria i. Neke atu i te te wā o rua tau, haere tūroro i te haamau'araa i te toharite o 25 ra i roto i te hōhipera i mua i te therapy ira tozero ra i roto i te hōhipera i muri.

Daniel Wheeler, he-tau 15-tau i Bristol nei ka mau wahi i roto i te tamataraa, mea kua puta ke te maimoatanga tona hauora. "Au pai ahau. maru ahau he rota iti ngāwari, e kore ahau i runga i hea tata rongoā rite maha, me te au whiwhi ahau atu o te mātauranga,"Ka mea ia.

"E kore matou e rite ki te whakamahi i te kupu 'rongoa', engari hoatu e i enei tūroro katoa mate nui, me te tohu, ngā tino nui whakatauria te reira i roto i te katoa o ratou,"Ka mea a Thrasher.

Tona rōpū e tumanako inaianei ki te tīmata tamataraa hou mō ngā tūroro ki te mate toronaihi pūtau me te thalassemia, e te hunga i roto i te riro ngā ira mate tino noa i roto i Ingarangi.

puta nga kaiputaiao ki te kua hinga i te raruraru nui i roto i te tahi mau tamataraa ira therapy o mua, te wahi i te ira whakaurua kino "ūnga atu" pānga, tae atu whakawhiti i runga i ira mate pukupuku-whakatairanga. I mua, etahi tamariki i homai ira therapy mo te tahi atu tikanga ārai, pērā i SCID (mirumiru syndrome pēpi), kua riro i runga ki whakawhanake kanisā ano te taha-pānga o te maimoatanga. "Ko te raruraru tiaki ratou ki te tahuri i runga i ētahi atu ira noa, tae atu ki ngā ira mate pukupuku,"Ka mea a Thrasher.

puta te tikanga hou ki te kua hinga tenei mā te whakauru i te wahi roa o te DNA, tikanga te ira noho i roto i te taiao ake taiao e puta ki te whakahaere i tona mahi atu tōtika.

Nga hua o te whakamatautauranga e "tino mīharo", rite ki Morris, nei te whai wāhi inaianei i roto i te toronga o te whakamatautauranga i e ngā tūroro pakeke mo te wa tuatahi. "Ko te he ara tino rerekē o te tuku maimoatanga ki tūroro,"Aded ia. "A, no te te raruraru ko te pūtau e kore e mahi tika, tuku ira therapy he ahua o te maimoatanga e kore e e taea ki te whakatutuki i taero me rongoā. "

whakaputaina te pūrongo i tenei ra i roto i te Journal o te American Medical Association.

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