Gene Therapy
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healthB.C. Family's Desperate Fight for Son's Gene Therapy
A British Columbia family is pleading for government assistance to access a life-saving gene therapy for their son, who suffers from a rare, aggressive neurodegenerative condition.
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healthEAHAD 2026: Ana Boban's Vision for European Haemophilia Care
Ana Boban, EAHAD President, outlines her goals to harmonize care, advance research, and support patients with inherited bleeding disorders.
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healthDavid Sinclair: Aging Is Reversible-Human Trials Begin Soon
Harvard geneticist David Sinclair says aging can be reversed using gene therapy, with human trials starting soon-potentially enabling lifespans into the 22nd century.
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healthEngineered Brain Cells Slash Alzheimer's Plaques by 50% in Mice
Breakthrough gene therapy reprograms astrocytes to clear amyloid beta, offering new hope for Alzheimer's treatment.
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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.