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Which of the following statements with regard to genetic medicine is/are correct?
1. Genetic medicines correct/compensate for the faulty genes responsible for disease.
2. Engineered viruses and lipid nanoparticles are used as carriers of the genetic medicine.
3. Genetic medicines alter the entire DNA sequence.
Select the answer using the code given below:
Explanation
Statement 1 is correct: Genetic medicines (such as gene therapy) are designed to treat or cure diseases by correcting, replacing, or compensating for faulty or mutated genes responsible for a disease.
Statement 2 is correct: To effectively deliver genetic material into a patient's cells, carriers or vectors are required. Engineered, harmless viruses (like adeno-associated viruses) and non-viral vectors like lipid nanoparticles (LNPs) are commonly used to safely transport the genetic medicine into the target cells.
Statement 3 is incorrect: Genetic medicines are highly targeted therapies. They aim to modify, delete, or replace specific genes or small sequences of DNA/RNA associated with a particular condition. They do not alter the entire DNA sequence (genome) of an individual.