gene therapy
18 stories
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healthPompe Disease: Breakthroughs in Early Detection and Advanced Therapies
New enzyme replacement, gene therapy, and digital monitoring are revolutionizing Pompe disease care, offering hope for earlier intervention and improved outcomes.
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healthUCLA Breakthrough: Gene Therapy Restores Cystic Fibrosis Function
UCLA researchers achieve gene therapy milestone, restoring full CFTR function in cystic fibrosis cells using non-viral nanoparticles.