A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the “bubble boy” disease.

A new gene therapy using patients’ own stem cells has safely cured ADA-SCID — the “bubble boy” disease — in 95% of children, restoring full immune function.

  • SaveTheTuaHawk@lemmy.caEnglish
    6·
    25 days ago

    The trick now is being able to get a commercial manufacturer to take the risk of developing this into an approved product

    Why do we need a commercial interest?

    NIH should do this. Too bad they were shit-canned by the Fascists.