Wilson's disease
Wilson's disease is a disease of abnormal copper ion metabolism. It is an autologous recessive disease. It is mainly caused by the mutation of the ATP7B gene in the 13th pair of chromosomes (pictured), resulting in the inability to synthesize proteins related to copper metabolism, and thus This causes copper ions to accumulate into cells and cause damage. Currently, the recommended treatments include supplementing zinc tablets, copper chelators, and dietary adjustments. There is no good cure yet. In recent years, gene therapy methods have become popular. Currently, there are foreign clinical trials using adenovirus vector (AAV9) containing the normal ATP7B gene, which is infused into the human body and affects abnormal cells to express the normal ATP7B gene. Currently, Taiwan does not have large-scale clinical trial plans. We hope that Taiwan can join relevant clinical trial plans in the future to benefit patients with Wilson's disease.
