CRISPR-Cas9 and Sickle Cell Anemia

The video discusses the CRISPR-based drug C Jevy, which targets sickle cell anemia by editing genes. It explains the genetic basis of the disease, the CRISPR mechanism, and how fetal hemoglobin can be used to alleviate symptoms. The video also covers the challenges and future potential of CRISPR technology in medicine.

What is the primary function of the CRISPR-Cas9 system?

• To produce proteins

• To transcribe RNA

• To cut and edit DNA

• To replicate DNA

Which gene is defective in sickle cell anemia?

• GAMMA gene

• ALPHA gene

• HBB gene

• BCL11A gene

What is the result of the single nucleotide polymorphism in the HBB gene?

• An increase in white blood cells

• A decrease in oxygen levels

• A change in the protein structure

• An increase in red blood cells

What does CRISPR stand for?

• Clustered Regularly Interspaced Short RNA Repeats

• Clustered Regularly Interspaced Short Palindromic Repeats

• Clustered Repeated Interspaced Short Palindromic Repeats

• Clustered Regularly Interspaced Short Protein Repeats

What is the role of the Cas9 enzyme in the CRISPR system?

• To replicate DNA

• To cut DNA

• To transcribe RNA

• To produce proteins

Which gene is targeted by CRISPR-Cas9 to treat sickle cell anemia?

• HBB gene

• BCL11A gene

• GAMMA gene

• ALPHA gene

What type of hemoglobin is produced by fetuses to receive oxygen?

• Beta hemoglobin

• Sickle hemoglobin

• Adult hemoglobin

• Fetal hemoglobin

What is one of the major challenges of CRISPR-Cas9 therapy?

• It requires stem cell transplantation

• It is inexpensive

• It is a simple process

• It can be done at any age

At what age must a patient be to receive the CRISPR-Cas9 therapy for sickle cell anemia?

• 18 years or older

• 6 years or older

• 12 years or older

• Any age

What is the estimated cost of the CRISPR-Cas9 therapy for sickle cell anemia?

• $3 million

• $500,000

• $1 million

• $2.2 million