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.

Statement 1 is CORRECT: Genetic medicines work by delivering genetic material (such as DNA or RNA) into a patient's cells to directly treat, prevent, or cure a disease. They achieve this by replacing or correcting a faulty, mutated gene, or by introducing a new gene to help the body compensate for a protein it cannot produce naturally.

Statement 2 is CORRECT: Naked genetic material cannot easily enter human cells on its own and is easily degraded by the body. To solve this, scientists use delivery vehicles (vectors). The most common carriers are:

Engineored viruses: (like Adeno-Associated Viruses or Lentiviruses) which are modified so they cannot cause disease but retain their ability to enter cells.

Lipid Nanoparticles (LNPs): Tiny fatty bubbles that encapsulate the genetic material, protecting it and helping it cross the cell membrane (famously used in mRNA COVID-19 vaccines).

Statement 3 is INCORRECT: Genetic medicines are highly targeted. They do not alter the entire DNA sequence of the human body. They only target the specific, localized gene or tissue responsible for the disease. For instance, gene therapies for hereditary blindness only target cells in the retina. Furthermore, many genetic medicines (like mRNA therapies) act temporarily in the cytoplasm without altering the patient's genomic DNA at all.