Autologous, Lentivirus-Modified, T Rapa Cell “Micropharmacies” for Lysosomal Storage Disorders

Scientists demonstrated that ex vivo lentivirus-modified, rapamycin-conditioned CD4+ T cells could also act as next-generation cellular delivery vehicles—that is, “micropharmacies”—to disseminate corrective enzymes for multiple lysosomal storage disorders.