The Fundació Per Amor a l'Art and the research center Médica Aplicada (CIMA ) of the University of Navarra have initiated a partnership to promote the study of Wilson's disease. The goal is to develop gene therapy treatments for this hereditary metabolic liver disease.

Wilson's disease is due to an alteration of copper metabolism that causes the accumulation of this metal in the liver and, subsequently, in other tissues such as the brain. It is caused by an inherited deficiency of the copper transporter ATP7B. It is a rare disease whose prevalence is between 1/30,000 and whose symptoms usually manifest in the first decade of life. Patients present with chronic hepatitis that can lead to cirrhosis, and when the disease affects the brain there are a wide variety of symptoms such as incoordination and tremors, speech difficulties, loss of report, decreased intellectual performance and many others. Currently there are treatments capable of eliminating the excess copper and which are effective in about 50% of patients, although the only curative treatment is liver transplantation.   

"We are very grateful to this Valencian entity, whose contribution will guarantee the preclinical development of a gene therapy treatment for Wilson's disease. The results obtained so far are very promising and are already in the patent process. Our goal is to take this drug to clinical application in the next three years", explains Dr. Gloria González Aseguinolaza, director of the Gene Therapy and Regulation of Gene Expression Program at CIMA and coordinator, together with Dr. Rubén Hernández, of this project initiated by Dr. Jesús Prieto.

Per Amor a l'Art Foundation

The Fundació Per Amor a l'Art is a Valencian entity that emerged as a continuation of a series of initiatives that began 10 years ago, aimed at helping others. Its mission statement is to awaken the sensibility of those whose conscience is asleep; to mobilize the conscience of those whose sensibility is awake; to share to help the most needy, especially children in status of vulnerability, to promote the research in orphan diseases, such as Wilson's disease, and to bring art closer to society.