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Retrovirus-mediated transfer of the human α- L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts

D. Kohn, X. Yu, E. Kakkis, 1997, Gene Therapy.

Expression of human Wiskott–Aldrich syndrome protein in patients’ cells leads to partial correction of a phenotypic abnormality of cell surface glycoproteins

S. Tsuboi, U. Francke, J. Derry, 2000, Gene Therapy.

Identification of Small Molecule Inhibitors of β-Amyloid Cytotoxicity through a Cell-Based High-Throughput Screening Platform

Marcie A. Glicksman, M. Michaelis, K. Seyb, 2008, Journal of biomolecular screening.