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.

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.

  • Yggstyle@lemmy.world
    19·
    22 hours ago

    Science

    …Although at the rate we are going it may well be referred to as such in the stories told about “the before times” as people huddle around the fire.

    • pelespirit@sh.itjust.works
      5·
      22 hours ago

      Magical science?

      Illnesses like KJ’s are the result of a single mutation — an incorrect DNA letter among the three billion in the human genome. Correcting it requires pinpoint targeting in an approach called base editing.

      To accomplish that feat, the treatment is wrapped in fatty lipid molecules to protect it from degradation in the blood on its way to the liver, where the edit will be made. Inside the lipids are instructions that command the cells to produce an enzyme that edits the gene. They also carry a molecular GPS — CRISPR — which was altered to crawl along a person’s DNA until it finds the exact DNA letter that needs to be changed.