Which of the following statements with regard to genetic medicine is/are correct? 1. Genetic medicin

Question

Which of the following statements with regard to genetic medicine is/are correct? 1. Genetic medicines correct/compensate for faulty genes responsible for disease. 2. Engineered viruses and lipid nanoparticles are used as carriers of genetic medicine. 3. Genetic medicines alter the entire DNA sequence.

Accepted Answer

Statement 1 is correct — genetic medicines treat disease at the root cause by correcting or compensating for faulty/missing genes. Statement 2 is correct — engineered viruses (AAV, lentiviruses) and lipid nanoparticles (LNPs) are the main delivery vectors. Statement 3 is incorrect — genetic medicines are highly precise, targeting only specific sequences/single genes, leaving the vast majority of the patient's ~3 billion base pairs unchanged.