An innovative platform approach for the development of ex-vivo gene therapies for the treatment of lysosomal storage diseases with skeletal involvement

C. Forni, M. Gabaldo,S. Crippa,S. Scala, G. Consiglieri,F. Tucci,L. Passerini,A. Gritti,S. Gregori,G. Ferrari, P. Albertini,S. Galimberti, M. G. Valsecchi, A. Corti, S. Zancan, M. Scarpa,R. Parini,L. Naldini,B. Gentner,M. E. Bernardo,A. Aiuti

HUMAN GENE THERAPY(2022)

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