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 :
Options
📖Detailed Explanation
Explanation:
Statement 1 is correct: The fundamental mechanism of action for genetic medicines (such as gene addition therapies, RNA therapeutics, and in vivo gene editing) is to treat conditions at their molecular source. They achieve this by either introducing a functional transgene to compensate for a non-functional or missing native gene, or by directly repairing a mutated locus to correct the faulty instruction responsible for cellular pathology.
Statement 2 is correct: Bare nucleic acids (DNA, mRNA, or siRNA) are highly unstable, prone to rapid enzymatic degradation by circulating nucleases, and cannot efficiently cross the hydrophobic, negatively charged cellular membrane. To solve this, specialized delivery systems are required.
Statement 3 is incorrect: Genetic medicines do not alter the entire DNA sequence of a human patient. The human genome is immense. Genetic therapies are designed to be highly precise, sequence-specific interventions.
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