Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1.
Human Gene Therapy
VanLith, Caitlin C; Guthman, Rebekah R; Nicolas, Clara T CT; Allen, Kari K; Du, Zeji Z; Joo, Dong Jin DJ; Nyberg, Scott L SL; Lillegard, Joseph B JB; Hickey, Raymond D RD