Comparative evaluation of genome editing and RNA interference for substrate reduction therapy in two animal models of glycogenosis
F. Collaud, P. Sellier, J. P. Parada-Zelada, S. Barral, M. Khoja,M. Gomez, N. Zabaleta,L. Van Wittenberghe,A. Miranda, B. Marolleau, G. Tanniou, F. Bordier, E. Bertil-Froidevaux, C. Georger,S. Jimenez, J. Cosette, N. Daniele,P. Colella, F. Mingozzi,G. Ronzitti HUMAN GENE THERAPY（2022）
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