Correct Option
The correct option is 1 and 2 only.
[as per provisional answerkey]Explanation
Genetic medicine is a branch of medical science that involves the use of genetic material (DNA or RNA) to treat, prevent, or cure diseases by addressing the underlying genetic cause rather than just managing symptoms.
- Statement 1 is Correct: Genetic medicines, including gene therapies, function by introducing a functional copy of a gene to compensate for a defective one, or by using molecular tools to silence or repair a faulty gene. This mechanism directly addresses the genetic mutations responsible for specific disorders.
- Statement 2 is Correct: To deliver genetic material into target cells, specialized delivery systems called vectors are required. Common vectors include engineered viruses (such as Adeno-associated viruses or Lentiviruses), which have been modified to remove their pathogenic properties, and non-viral systems like lipid nanoparticles (LNPs), which encapsulate the genetic material for safe transport through the bloodstream.
- Statement 3 is Incorrect: Genetic medicines are highly targeted and do not alter the entire DNA sequence of an organism. They are designed to modify, replace, or supplement specific genes or sequences associated with a particular disease. Broad, non-specific alteration of the entire genome is neither the objective nor the capability of current genetic medicine technologies.
Key Takeaway: Genetic medicine utilizes specific delivery vectors (viral or non-viral) to target and correct localized genetic defects without modifying the organism's entire genome.