Understanding Blood Disorders and Gene Therapy

What is the primary function of red blood cells in the body?

• To produce hormones

• To control blood clotting

• To supply oxygen to cells and tissues

• To fight infections

Which gene mutation is responsible for beta thalassemia?

• CFTR gene

• TP53 gene

• BRCA1 gene

• HBB gene

What is a common symptom of sickle cell disease?

• Improved vision

• Severe pain episodes

• Increased appetite

• Rapid hair growth

What is the role of hematopoietic stem cells in gene therapy?

• To produce insulin

• To generate nerve cells

• To form new skin cells

• To create red and white blood cells and platelets

How is the working gene delivered into the hematopoietic stem cells during ex vivo gene therapy?

• By direct injection into the bloodstream

• Via a viral vector

• Using a chemical compound

• Through a bacterial vector