Question
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.
Explanation
Genetic medicines represent a shift from treating only symptoms to addressing diseases at the genetic level. Depending on the therapy, they can replace, modify or regulate specific genetic material using specialised delivery systems.
Statement 1 — Correct: Genetic medicines, including gene therapy and gene editing, aim to treat or prevent diseases by addressing their underlying genetic cause. They may add, remove or modify genetic material to correct or compensate for faulty genes.
Statement 2 — Correct: Genetic material generally requires a delivery vector to enter target cells. These include engineered viruses such as adeno-associated viruses and lentiviruses, as well as non-viral systems such as lipid nanoparticles (LNPs), which can deliver genetic payloads into cells.
Statement 3 — Incorrect: Genetic medicines do not alter the entire DNA sequence of a person. Most therapies target a specific gene or genetic sequence, leaving the vast majority of the patient’s DNA unchanged.
Therefore, Statements 1 and 2 are correct.