Gene Therapy: Correcting Genetic Defects — NEET Biology
✅ Asked in NEET 2021If a person is born with a hereditary disease, can a corrective therapy be taken for such a disease? Gene therapy is an attempt to do this. Gene therapy is a collection of methods that allows correction of a gene defect that has been diagnosed in a child/embryo. Here genes are inserted into a person's cells and tissues to treat a disease. Correction of a genetic defect involves delivery of a normal gene into the individual or embryo to take over the function of and compensate for the non-functional gene.
Gene therapy is the clinical application of inserting a normal, functional gene into a patient's cells to compensate for a defective or non-functional gene causing hereditary disease. Students often confuse gene therapy with gene editing (like CRISPR, which modifies the defective gene itself)—remember that gene therapy delivers a *normal replacement gene* rather than fixing the faulty one. The key point NTA tests is understanding that this correction can happen in children or embryos, and it works by allowing the normal gene to take over the function. To avoid mistakes, focus on the delivery mechanism and functional compensation, not gene repair. This appears in biotechnology applications, a high-yield NEET topic testing real-world genetic medicine applications.
When gene targeting involving gene amplification is attempted in an individual's tissue to treat disease, it is known as: (NEET 2021)
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