The future of gene-targeted therapy for hereditary tyrosinemia type 1 as a lead indication among the inborn errors of metabolism.

Thompson, Whitney S; Mondal, Gourish; Vanlith, Caitlin J; Kaiser, Robert A; Lillegard, Joseph B.

Afiliação

Thompson WS; Department of Pediatrics, Mayo Clinic, Rochester, MN, USA.
Mondal G; Department of Surgery, Research Scientist, Mayo Clinic, Rochester, MN, USA.
Vanlith CJ; MD/PhD student, Mayo Clinic, Rochester, MN, USA.
Kaiser RA; Department of Surgery, Research Scientist, Mayo Clinic, Rochester, MN, USA.
Lillegard JB; Midwest Fetal Care Center, Childrens Hospital of Minnesota, MN, USA.

Expert Opin Orphan Drugs ; 8(7): 245-256, 2020.

Article em En | MEDLINE | ID: mdl-33224636