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Johns Hopkins

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Medicine

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Robert A. Kaiser 1,2, Shennen A. Mao 1, Jaime Glorioso 3, Bruce Amiot 1, Clara T. Nicolas 1, Kari L. Allen 1, Zeji Du 1, Caitlin J VanLith 1, Raymond D. Hickey 1, Scott L. Nyberg 1, Joseph B. Lillegard 1,2,4
1Department of Surgery, Mayo Clinic, 2Midwest Fetal Care Center, Children's Hospitals and Clinics of Minnesota, 3Department of Surgery, Johns Hopkins, 4Pediatric Surgical Associates

This protocol is intended to describe porcine hepatocyte isolation and ex vivo gene delivery to cure models of metabolic diseases via autologous cell transplantation. Although this particular model enjoys unique advantages that favor successful therapy, the application is a relevant foundation to address additional diseases and indications.

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