Changing DNA in a Cell With No DNA: Gene Therapy for Blood Disorders

What is the main idea behind gene therapy?

• To modify genetic code to treat or cure diseases

• To create new genes for better health

• To eliminate all genetic disorders

• To enhance human physical abilities

What is the primary cause of sickle cell disease?

• An autoimmune disorder

• A structural change in hemoglobin

• A viral infection

• A deficiency in red blood cells

What is a major challenge in using viruses to insert beta-globin genes?

• Viruses are too expensive to produce

• Viruses may insert genes in incorrect locations

• Viruses may cause other diseases

• Viruses cannot replicate

Why is editing the beta-globin gene directly considered effective?

• It is cheaper than other methods

• It avoids the risk of side effects by knowing the exact location of changes

• It requires no clinical trials

• It adds new genes to the genome

What is the alternative strategy involving gamma-globin?

• Replacing gamma-globin with beta-globin

• Reactivating the gamma-globin gene by deactivating its regulatory gene

• Using gamma-globin to produce more red blood cells

• Creating new gamma-globin genes

What potential advantage does reactivating gamma-globin have over other treatments?

• It is faster to implement

• It requires no genetic modification

• It can treat multiple blood disorders

• It is less effective but cheaper

Which blood disorder, besides sickle cell, might benefit from gamma-globin reactivation?

• Leukemia

• Hemophilia

• Beta-thalassemia

• Anemia