The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.

cross-posted from: https://slrpnk.net/post/22218016

The work, they said, began decades ago with federal funding for basic research on bacterial immune systems. That led eventually, with more federal support, to the discovery of CRISPR. Federal investment in sequencing the human genome made it possible to identify KJ’s mutation. U.S. funding supported Dr. Liu’s lab and its editing discovery. A federal program to study gene editing supported Dr. Musunuru’s research. Going along in parallel was federally funded work that led to an understanding of KJ’s disease.

  • k0e3@lemmy.caEnglish
    10·
    8 days ago

    Is a mutated gene carried by all cells in the body? Even if that’s the case, do doctors only target the affected area (the liver, for KJ) because that’s the only part of the body where the funky gene will get revealed (is that the right term)?

    I know what gene editing is, but I’m still trying to wrap my head around how that’s used to treat people. Such amazing work by the doctors and FDA.

      • k0e3@lemmy.caEnglish
        1·
        7 days ago

        Right so when they treat a disease with gene therapy, they don’t need to fix every cell in the body, right? They just target the area affected? Does that mean you can still pass it on even if you’re “cured”?

  • Sterile_Technique@lemmy.worldEnglish
    6·
    7 days ago

    Personalized gene editing isn’t new, is it? IIRC they did this successfully for sickle cell anemia or something; but it’s stupid expensive, so if you’re not crazy rich you don’t have access to this kind of healthcare.