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Description
Most prominently, studies on RTD caused by SLC52A2/SLC52A3 mutations show that systemic delivery of AAV9-SLC52A2 in loss-of-function mouse models restores transporter expression, corrects spinal motor neuron energy deficits, reverses motor dysfunction, and extends survival
doi: 10.34067/KID.0006672021
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Sardina, J
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