Correction of Gb3 levels in alpha-Galactosidase A deficient mice by enzyme produced in vivo by shielded engineered cells

E. Makino, B. Fluharty, D. Tietz,M. Donovan, E. Pearson,L. Barney,J. Sewell, M. McAulife, S. Shrestha,J. Huang,R. Newman, O. O'Conner, T. Glyptis, C. Sparages, V. Zhao, S. Brittes Boss, K. Barret, M. Gorgievski,D. Moller, D. Gorzo, D. Peritt, R. Vivaldi

HUMAN GENE THERAPY(2019)

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