In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery

Breuer, CB; Hanlon, KS; Natasan, JS; Volak, A; Meliani, A; Mingozzi, F; Kleinstiver, BP; Moon, JJ; Maguire, CA

Moon, JJ (corresponding author), Massachusetts Gen Hosp, Ctr Immunol & Inflammatory Dis, Charlestown, MA 02129 USA.; Maguire, CA (corresponding author), Massachusetts Gen Hosp, Dept Neurol, Charlestown, MA 02129 USA.; Moon, JJ (corresponding author), Massachusetts Gen Hosp, Div Pulm & Crit Care Med, Charlestown, MA 02129 USA.; Moon, JJ; Maguire, CA (corresponding author), Harvard Med Sch, Boston, MA 02115 USA.

SCIENTIFIC REPORTS, 2020; 10 (1):

Abstract

Ex-vivo gene therapy using stem cells or T cells transduced by retroviral or lentiviral vectors has shown remarkable efficacy in the treatment of immu......

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