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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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Exome sequences from HCC lines were mapped to reference human genome sequences using GATK
Long-term risks have not been identified
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