Gene Editing and Sickle Cell Treatment

The video discusses the use of CRISPR, a gene-editing tool, in treating sickle cell disease. It highlights the approval of Casgevy, a CRISPR-based therapy, and Lyfgenia, which uses lentiviral vectors. The video explains the mechanisms of these treatments, their effectiveness, and the challenges faced, including high costs and limited accessibility. It emphasizes the potential of CRISPR in medicine and the beginning of its application in patient care.

What is the primary function of CRISPR in gene editing?

• To delete entire chromosomes

• To replicate DNA sequences

• To precisely cut DNA at specific locations

• To create new genes

Which of the following is NOT a symptom of sickle cell disease?

• High blood pressure

• Fever

• Vaso-occlusive crises

• Anemia

What is the main advantage of Casgevy over traditional bone marrow transplants?

• It can be done at home

• It is a one-time treatment

• It is less expensive

• It requires no donor match

How does Casgevy help in treating sickle cell disease?

• By removing sickle-shaped cells

• By altering the shape of adult hemoglobin

• By reactivating fetal hemoglobin production

• By increasing red blood cell count

What technology does Lyfgenia use for gene editing?

• TALENs

• Zinc finger nucleases

• CRISPR-Cas9

• Lentiviral vectors

What is a potential risk associated with Lyfgenia treatment?

• Permanent alteration of all body cells

• Development of acute myeloid leukemia

• Immediate allergic reactions

• Increased risk of diabetes

What is a major challenge in accessing CRISPR-based treatments like Casgevy?

• Unavailability of the technology

• Lack of trained medical professionals

• Lack of patient interest

• High cost and limited treatment centers

What is the estimated cost of Casgevy treatment?

• 2.2 million US dollars

• 4 million US dollars

• 1 million US dollars

• 3.1 million US dollars

What is a common requirement for both Casgevy and Lyfgenia treatments?

• Chemotherapy to eliminate faulty stem cells

• Immediate results post-treatment

• Editing of all body cells

• No need for chemotherapy

What is a significant benefit observed in patients after receiving Casgevy treatment?

• Complete cure of all genetic disorders

• No more hospital visits for sickle cell crises

• Increased immunity to all diseases

• Permanent change in DNA structure