Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria

C. O. Harding, M. B. Gillingham, K. Hamman, H. Clark, E. Goebel-Daghighi, A. Bird, D. D. Koeberl

Research output: Contribution to journal › Article › peer-review

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Medicine & Life Sciences