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gene replacement therapy

Coverage of gene replacement therapy in the Nexus archive.

Earliest in view: May 20 · 18:48 UTCMost recent: May 20 · 18:48 UTC
Co-mentioned in this coverage
Tippi MacKenzieperson1Food and Drug Administrationorganization1STAT+organization1San Franciscoplace1lysosomal storage disordertopic1
Recent coverage
  • HEALTHMay 20 · 18:48 UTCSTAT NEWS
    STAT+: Pioneering trial for treating genetic disease before birth nears reality

    Tippi MacKenzie's team has submitted an application to the FDA for a trial to treat a rare genetic disease in fetuses using gene replacement therapy. The trial aims to treat five fetal patients with a lysosomal storage disorder. If approved, this would be a significant step towards treating genetic diseases before birth.