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Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disorders
Murtaza S Nagree 1 2, Tania C Felizardo 3, Mary L Faber 2, Jitka Rybova 2, C Anthony Rupar 4, S Ronan Foley 5, Maria Fuller 6, Daniel H Fowler 3, Jeffrey A Medin 1 2 7
...We demonstrate that ex vivo lentivirus-modified, rapamycin-conditioned CD4+ T cells can also act as next-generation cellular delivery vehicles-that is, "micropharmacies"-to disseminate corrective enzymes for multiple lysosomal storage disorders. We evaluated the therapeutic potential of this treatment platform for Fabry, Gaucher, Farber, and Pompe diseases in vitro and in vivo. For example, such micropharmacies expressing α-galactosidase A for treatment of Fabry disease were transplanted in mice where they provided functional enzyme in key affected tissues such as kidney and heart, facilitating clearance of pathogenic substrate after a single administration.