Blindfödda barn fick syn efter genterapi

Elva små barn som föddes med kraftigt nedsatt syn, och i juridisk mening var blinda, fick alla synskärpa efter behandling med en ny genterapikandidat.

By Anders Göransson

February 24, 2025 11:05 AM

I en artikel, som publicerats i The Lancet, redovisas resultat för fyra av barnen, som behandlats med en genterapi utvecklad av amerikanska MeiraGTx. Barnen, som var mellan 1 och 2,8 år vid behandlingens början, hade LCA4, en sällsynt form av Retinitis pigmentosa, som är en grupp ärftliga sjukdomar som påverkar näthinnans synceller.

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