Here the authors report the engineering of human red blood cells with an entire metabolic pathway as a potential strategy to treat patients with guanidinoacetate methyltransferase (GAMT) deficiency, capable of reducing the high toxic levels of guanidinoacetate acid (GAA) and restoring proper creatine levels in blood and tissues. We first produced a recombinant form of […]
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Degradation of GAA in engineered cells
- May 9, 2022
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