Gene Therapy Cures Immune System Defects

The video discusses Mustafa Hassan, a child born without a working immune system, who was successfully treated with gene therapy. This experimental technique repaired his inherited defect, allowing him to live a normal life. The process involves using a virus to deliver a healthy gene into bone marrow cells, which are then reintroduced into the patient. The therapy has been successful in 14 children, with only two failures, and has saved the NHS significant costs. Despite past concerns about cancer risks, improvements have made the therapy safer, and it is now considered part of mainstream medicine.

What was the main health issue faced by Mustafa Hassan?

• He suffered from a rare blood disorder.

• He had a chronic lung disease.

• He was born without a working immune system.

• He was born with a defective heart.

What is the first step in the gene therapy process?

• Isolating the defective gene.

• Taking a bone marrow sample from the child.

• Using a virus to deliver a healthy gene.

• Injecting healthy cells into the child.

How do doctors use a virus in the gene therapy process?

• To prevent infections in the body.

• To destroy the defective cells.

• To carry a healthy gene into bone marrow cells.

• To boost the immune system directly.

What was a major concern with gene therapy trials a decade ago?

• The high cost of the treatment.

• The risk of activating cancer genes.

• The long recovery time for patients.

• The lack of qualified doctors.

What has Mustafa's recovery demonstrated about gene therapy?

• It is a temporary solution.

• It is only effective in children.

• It is now considered part of mainstream medicine.

• It is still an experimental technique.