Rescued by fat bubbles: Scientists treat rare genetic disease with designer molecule

DALLAS — Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 antitrypsin deficiency — a promising leap toward treating humans with this rare inherited disease. In a paper published last month in the journal Nature Biotechnology, researchers at UT Southwestern Medical Center introduced lipid nanoparticles — hollow, fatty spheres ...