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Forskare vid Harvard i Boston och universitetet i Paris har för första gången lyckats bota en patient med den svåra blodsjukdomen Thalassemi genom genterapi. Den genetiska sjukdomen uppstår då globingenerna, generna som kodar för en viktig beståndsdel i röda blodkroppar, inte fungerar eller saknas helt.
Patienten var en 18-årig man som sedan barnsben levt med sjukdomen som gör att han måste genomgå blodtransfusioner varje månad. För tre år sedan tog forskarna ut stamceller från patientens benmärg. Forskarna injicerade sedan in friska globingener i lentivirus som i sin tur fördes in i stamcellerna. De behandlade stamcellerna kom åter till patienten och
redan ett år efter behandlingen hade hans blod nått sådana halter av röda blodkroppar att han inte längre behövde genomgå blodtransfusioner. Idag, tre år efter behandlingen är han fortfarande symtomfri.
Behandlingen beskrivs i tidsskriften Nature.
Patienten var en 18-årig man som sedan barnsben levt med sjukdomen som gör att han måste genomgå blodtransfusioner varje månad. För tre år sedan tog forskarna ut stamceller från patientens benmärg. Forskarna injicerade sedan in friska globingener i lentivirus som i sin tur fördes in i stamcellerna. De behandlade stamcellerna kom åter till patienten och
redan ett år efter behandlingen hade hans blod nått sådana halter av röda blodkroppar att han inte längre behövde genomgå blodtransfusioner. Idag, tre år efter behandlingen är han fortfarande symtomfri.
Behandlingen beskrivs i tidsskriften Nature.
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