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. Select the answer using the code given below :

Correct Option: C (1 and 2 only)

Statement 1 is correct: According to the U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH), genetic medicine (or gene therapy) aims to treat or cure diseases by modifying, manipulating, or replacing a patient's genetic makeup. It traditionally works by introducing a healthy copy of a gene, inactivating a mutated gene, or compensating for faulty genes to restore normal protein function.

Statement 2 is correct: Naked genetic material cannot efficiently enter human cells or survive bodily defenses on its own. Therefore, it requires delivery vehicles known as vectors. Engineered viruses (such as adeno-associated viruses or lentiviruses) are commonly used because of their natural, evolutionary ability to infect cells and deliver genetic material. However, because viral vectors can sometimes trigger immune responses, non-viral carriers like lipid nanoparticles (LNPs) are also widely utilized. LNPs encapsulate the genetic payload to safely deliver it across the cell membrane, a technology famously utilized in the 2020 mRNA COVID-19 vaccines and increasingly in modern CRISPR therapies.

Statement 3 is incorrect: Genetic medicines do not alter the entire DNA sequence. The human genome contains roughly 3 billion base pairs. Genetic therapies are designed to be highly precise; advanced techniques like CRISPR-Cas9 or base editing target only the specific faulty gene or point mutation responsible for the disease. The rest of the genome is left untouched. Altering the entire DNA sequence is medically unnecessary and scientifically impossible.

Takeaway: Think of genetic medicine as a highly precise "find and replace" function in a word processor. It targets a specific typo (the faulty gene) using a customized delivery envelope (viral or lipid vectors), rather than rewriting the entire book (the entire DNA sequence).