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Gene therapies for blood and clotting disorders could add years of life for terminal conditions and relief from lifelong, debilitating flare-ups and hospitalizations.[1, 2] Many CGTs offer the prospect of one-time or short-term treatment that produces long-lasting improvements

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Furthermore, the pQCT findings hint that the noted increase in cortical BMC could be due to an expansion in the bone's cross-sectional area

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