Understanding Wilson Disease and Gene Therapy

What is the primary cause of Wilson disease?

• High levels of calcium

• Mutations in the ATP-7B gene

• Excessive iron in the body

• Lack of vitamin D

Which of the following is a common symptom of advanced liver dysfunction in Wilson disease?

• Increased appetite

• Improved vision

• Chronic fatigue

• Weight gain

How do current treatments for Wilson disease primarily work?

• By reducing calcium levels

• By lowering copper to non-toxic levels

• By increasing copper levels

• By enhancing iron absorption

What is the goal of gene therapy in treating Wilson disease?

• To enhance vitamin D production

• To remove all copper from the body

• To deliver a working ATP-7B gene

• To increase copper absorption

What method is used to deliver the gene therapy for Wilson disease?

• Oral medication

• Inhalation

• Intravenous infusion

• Topical application