Sickle Cell Disease and Gene Therapy

Sickle cell disease is a genetic blood disorder causing red blood cells to become sickle-shaped, leading to various health issues like pain and organ damage. Despite advancements in care, life expectancy remains significantly reduced. Recent research, including the Edit 301 trial, offers hope through gene editing using CRISPR technology. This involves modifying stem cells to produce healthy hemoglobin, potentially providing a functional cure. The treatment process includes chemotherapy and stem cell infusion, aiming to prevent future damage while not reversing existing effects.

What is the primary genetic defect in sickle cell disease?

• A defect in the genes that make red blood cells

• A defect in white blood cells

• A defect in the nervous system

• A defect in the immune system

How does the shape of red blood cells change in sickle cell disease?

• They become square-shaped

• They become triangular

• They become sickle-shaped

• They remain circular

What is a common complication of sickle cell disease?

• Severe pain crises

• Hearing loss

• Vision problems

• Frequent headaches

What is the average life expectancy for patients with sickle cell disease in the United States?

• Around 43 years

• Around 50 years

• Around 60 years

• Around 70 years

What is the goal of the Edit 301 clinical trial?

• To create a vaccine for sickle cell disease

• To improve surgical techniques

• To develop a new pain medication

• To use gene therapy to treat sickle cell disease

What technology is used in the Edit 301 trial to modify genes?

• Gene splicing

• CRISPR

• RNA interference

• Gene cloning

What is the role of CRISPR in the Edit 301 trial?

• To enhance immune response

• To increase red blood cell count

• To precisely modify specific genes

• To remove damaged cells

What is the purpose of chemotherapy in the treatment process?

• To reduce pain

• To enhance gene therapy

• To remove old bone marrow

• To increase white blood cell count

How are the modified stem cells introduced into the patient's body?

• Via a surgical procedure

• As a blood transfusion

• Through oral medication

• Through inhalation

What is the ultimate goal of the gene therapy discussed in the video?

• To reduce the frequency of hospital visits

• To improve the quality of life temporarily

• To completely eliminate sickle cell disease

• To achieve a functional cure and prevent future damage