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Genetic hepatic disease
SCIE.05.158
AGE OF ADMINISTRATION IMPACTS THE EFFICIENCY OF LENTIVIRAL VECTOR-MEDIATED HEPATOCYTE TRANSDUCTION IN VIVO AND ITS DISTRIBUTION IN THE LIVER LOBULE

Starinieri F. * , Milani M. , Beretta S. , Canepari C. , Simoni C. , Fabiano A. , Cammarota E. , Biffi M. , Russo F. , Merelli I. , Cantore A.

Milan
SCIE.05.159
MOLECULAR MECHANISMS COORDINATING MEMBRANE TRAFFICKING AND ION TRANSPORT IN WILSON DISEASE

Polishchuk E. * [1] , Petruzzelli R. [1] , Catalano F. [1] , Crispino R. [1] , De Cegli R. [1] , Zischka H. [2] , Di Schiavi E. [3] , Polishchuk R. [1]

Pozzuoli [1] , Munich [2] , Naples [3]
SCIE.11.160
NUCLEASE-FREE TARGETED INTEGRATION OF A PROMOTERLESS MINI-ATP7B CONFERS PROLIFERATIVE ADVANTAGE TO EDITED HEPATOCYTES AND CORRECTS WILSON DISEASE

Padula A. [1] , Spinelli M. [2] , Nusco E. [1] , Capolongo F. [1] , Campione S. [2] , Perna C. [1] , Bastille A. [3] , Ericson M. [3] , Wang C. [3] , Zhang S. [3] , Amoresano A. [2] , Nacht M. [3] , Piccolo P. * [1]

Pozzuoli [1] , Napoli [2] , Lexington, MA [3]
SCIE.05.161
MODELING WOLMAN DISEASE USING GENETICALLY ENGINEERED HUMAN LIVER ORGANOIDS

Anfuso B. [1] , Selvestrel D. [1] , Altieri A. [1] , Mattivi A. [2] , Fava L. [2] , Sorrentino G. * [1] , Conti L. [2]

Trieste [1] , Trento [2]