TY - JOUR AU - Schilsky, Michael L AU - To, Uyen AU - Camarata, Michelle A. PY - 2018 DA - 2018/08/22 TI - Prospects for Cure in Wilson Disease JO - OBM Genetics SP - 030 VL - 02 IS - 03 AB - Wilson Disease is a monogenetic disorder of copper metabolism affecting the ATP7B gene. Treatment is lifelong and focuses on removal of copper to arrest disease progression and improve the clinical manifestations of copper toxicity. Currently the only cure is replacement of ATP7B in hepatocytes by liver transplantation, however, lifelong monitoring and immunosuppressive medications are still needed. The possibility of introducing functional ATP7B gene through gene therapy provides an exciting potential option for achieving a more permanent cure without the need for additional therapy and medical monitoring. SN - 2577-5790 UR - https://doi.org/10.21926/obm.genet.1803030 DO - 10.21926/obm.genet.1803030 ID - Schilsky2018 ER -