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

The video discusses gene therapy's potential to treat genetic diseases, focusing on sickle cell disease. It explains the challenges and advancements in gene therapy, including the use of lentivirus and gene editing techniques. The video also explores the reactivation of gamma-globin as a promising treatment for sickle cell and other blood disorders. Despite the challenges, ongoing research offers hope for effective treatments.

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