Pre-clinical development of an ex-vivo Hematopoietic Stem/progenitor Cells-gene therapy for a-Mannosidosis

P. Quaranta, A. A. Rilievo, L. Basso-Ricci,R. J. Hernandez, C. Forni, I. Visigalli, P. Albertini, A. Paoli,A. Morrone,R. Parini, B. Gentner, S. Crippa,M. E. Bernardo,S. Scala,A. Aiuti

HUMAN GENE THERAPY(2022)

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