NATIONAL QUARTERFINAL STAGE 2026
St. John's School: 43
Saviour SHS: 33
Mfantsiman Girls' SHS: 2
QUESTION
What is the main aim of gene therapy?
ANSWER: The aim is to cure (treat) genetic diseases.
EXPLANATION
Gene therapy works by introducing, removing, or altering genetic material within a patient's cells to correct the effects of a faulty or missing gene, with the overall goal of treating or curing inherited genetic diseases (e.g. cystic fibrosis, sickle cell disease) at their root cause rather than just managing symptoms.
PRACTICE QUESTIONS
1. What is the main aim of gene therapy?
ANSWER: To treat or cure genetic diseases
2. How does gene therapy work?
ANSWER: By introducing, removing or altering genes in a patient's cells
3. What is a vector in gene therapy?
ANSWER: A carrier, often a modified virus, that delivers the gene
4. Name a virus used as a vector.
ANSWER: Adenovirus (or retrovirus)
5. What is somatic gene therapy?
ANSWER: Treating body cells, so the change is not inherited
6. What is germline gene therapy?
ANSWER: Changing egg or sperm cells so the change is inherited
7. Name a genetic disease that gene therapy has been used against.
ANSWER: Sickle cell disease (or cystic fibrosis, SCID)
8. Which gene is faulty in cystic fibrosis?
ANSWER: CFTR gene
9. Name a gene editing tool.
ANSWER: CRISPR-Cas9
10. What is ex vivo gene therapy?
ANSWER: Cells are modified outside the body and returned
11. State one risk of gene therapy.
ANSWER: An immune reaction to the vector
12. What is a genetic disease?
ANSWER: A disease caused by a faulty gene or chromosome change