Preclinical Workup for an Approved Phase I/II Clinical Trial for Mucopolysaccharidosis II (MPSII): Validation of a GMP Stem Cell Gene Therapy Manufacturing Process

Stuart M. Ellison,Karen Buckland, Yuko Learmonth, Francisco R. Rodriguez, Jose B. Arago,Simon Jones,Robert Wynn,Claire Booth,Brian W. Bigger

MOLECULAR THERAPY(2023)

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