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

DALLAS, TEXAS, JUL 19 – UT Southwestern researchers used lipid nanoparticles to correct gene mutations in mice, restoring 80% of normal liver function and 90% of lung function in alpha-1 antitrypsin deficiency models.

Summary by Medical Xpress
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.

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The Union Democrat broke the news in on Friday, July 18, 2025.
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