Filippo Carlucci

TL;DR: Sustained engraftment of engineered HSCs with differentiation into multiple lineages resulted in increased lymphocyte counts, improved immune functions, and lower toxic metabolites, indicating the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID.

TL;DR: Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible.

TL;DR: Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement is shown to occur after two weeks after treatment with PBL.

TL;DR: This investigation investigated the medium-term outcome of gene therapy (GT) in 18 patients with ADA-SCID for whom an HLA-identical family donor was not available; most were not responding well to ERT.

TL;DR: MP at 20°C improves cell survival and leads to a marked improvement in hepatic preservation of steatotic livers as compared with cold storage, substantiated by better morphology, higher glycogen content, and lower reactive oxygen species production by sinusoidal cells in Steatotic liver submitted to MP at20°C versus cold storage.