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A gene therapy pipeline takes shape for a cluster of rare diseases
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A gene therapy pipeline takes shape for a cluster of rare diseases

 Danielle Ternes/BioPharma Dive AUTHOR Ned Pagliarulo@NedPagliarulo PUBLISHED Feb. 12, 2021 The toxic globules of sugar and fat slowly pile up in the body’s cells, accumulating in blood vessels and in major organs like the kidney and heart. Typically, special-purpose proteins within the cell would tear apart and break down the toxins. But for people with...

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