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Wang, Y.-P
10.1038/ncpneph0655 152 VanholderR.De SmetR.GlorieuxG.ArgilsA.BaurmeisterU.BrunetP.et al (2003)
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
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