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Gene Therapy Technique Slows Brain Disease (1 of 2)

Caption: Progeny of HSCs that were engineered to carry the correct version of a gene (through the integration of a lentiviral vector) distribute throughout the body. Cartier et al. show that some cells replaced diseased microglia in the brain and relieved lipid storage in patients suffering from ALD.

Credit: Image courtesy of Y. Greenman/Science

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