Which of the following statements with regard to genetic medicine is/are correct ?
- Genetic medicines correct/compensate for the faulty genes responsible for disease.
- Engineered viruses and lipid nanoparticles are used as carriers of the genetic medicine.
- Genetic medicines alter the entire DNA sequence.
Select the answer using the code given below :
Detailed Explanation:
Statement 1 — Correct. Genetic medicines (gene therapy and gene editing) treat diseases at their root cause by introducing, removing, or changing genetic material to correct or compensate for faulty/missing genes responsible for a disease.
Statement 2 — Correct. Genetic material cannot be injected directly — it requires a vector (carrier):
| Vector Type | Example | Mechanism |
|---|---|---|
| Engineered Viruses | AAV, Lentiviruses | Modified to remove disease-causing properties; deliver therapeutic genes |
| Lipid Nanoparticles (LNPs) | mRNA/CRISPR delivery | Encapsulate genetic payload in protective fat layer |
Statement 3 — Incorrect. Genetic medicines do NOT alter the entire DNA sequence. The human genome has ~3 billion base pairs and 20,000+ genes. Gene therapies are highly precise — targeting only specific localized sequences or single genes while leaving the vast majority of native DNA completely unchanged.
Key Trick: Statement 3 uses the word "entire" — a classic UPSC exaggeration trap. Precision, not wholesale alteration, is the hallmark of genetic medicine.
Question 1 of 5 Gene Therapy and CRISPR
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Consider the following statements: Genetic changes can be introduced in the cells...