AAV9-Mediated Gene Therapy for NGLY1 Deficiency and Assessment of GNA Biomarker Changes in a Rat Disease Model Mueller, W. F., Zhu, L., Dwight, S. S., Beahm, B., Lee, K. J., Wilsey, M., Mak, J., Pollard, L., Wood, T., Cowan, T., Crawford, B., Wechsler, T. CELL PRESS. 2020: 124–25

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