The First CRISPR Gene Therapy Is Here

What blood disorders can be treated with Casgevy, the first CRISPR-Cas9 gene therapy?

• Hemophilia and Leukemia

• Sickle Cell Disease and β-Thalassemia

• Anemia and Jaundice

• Malaria and Stroke

What causes the red blood cells of a person with Sickle Cell Disease to change shape?

• A viral infection that attacks blood cells.

• A mutation in both copies of the gene for hemoglobin.

• Lack of iron in the blood.

• An overproduction of white blood cells.

How does the Cas9 enzyme, used in CRISPR gene editing, function?

• It helps blood cells carry more oxygen.

• It acts like molecular scissors to cut DNA at specific points.

• It strengthens the immune system against viruses.

• It replaces damaged cells with healthy ones.

How does the gene therapy Casgevy help treat Sickle Cell Disease?

• It directly corrects the mutation in the adult hemoglobin gene.

• It introduces a new, healthy adult hemoglobin gene into the cells.

• It silences a gene that normally turns off fetal hemoglobin production.

• It replaces all the sickle-shaped red blood cells with normal ones.

What is a potential problem with using lentiviruses to deliver new genes into cells?

• They always cause allergic reactions.

• They can insert genes in random places, possibly causing cancer.

• They only work on certain types of cells.

• They are too large to enter human cells.

What is a known risk associated with Lyfgenia treatment for sickle cell disease?

• It can cause patients to develop a new type of anemia.

• It requires patients to take daily medication for life.

• It carries a "black box warning" for lifelong blood cancer monitoring.

• It makes patients immune to all other diseases.

Which of these steps is part of the gene editing treatment process for sickle cell disease?

• Taking a daily pill to replace faulty blood cells.

• Undergoing chemotherapy to remove existing bone marrow.

• Receiving a single injection that cures the disease instantly.

• Eating a special diet to boost stem cell production.

What is a major challenge in making gene editing treatments for sickle cell disease widely available?

• The treatments are not very effective.

• There are not enough patients who need the treatment.

• The high cost makes them inaccessible to many people.

• The technology is still in early experimental stages.