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How does gene therapy aim to treat inherited genetic diseases?

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BRCA2 tumor suppressor gene on chromosome 13 — human genetic disease Photomicrograph of the *BRCA2* tumor suppressor gene on chromosome 13 of the human genome. Inactivation of this growth-regulating gene is associated with a higher risk of developing breast cancer.

Gene therapy aims to treat inherited genetic diseases by introducing a normal gene into an individual’s genome to repair a disease-causing mutation. When a normal gene is successfully inserted into the nucleus of a mutant cell, it may replace the defective allele or integrate into the genome, allowing the cells to produce the necessary gene product to restore a healthy phenotype.1

The process typically requires a delivery system, often a modified virus known as a viral vector, which efficiently transports the therapeutic genetic material into target cells. Researchers are also exploring stem cell therapy, where cells are reprogrammed and corrected before being reintroduced to the body.12

While somatic cell therapy treats the individual, it does not pass modifications to future generations. Conversely, germline gene therapy aims to alter reproductive cells, which would be heritable; however, this remains experimental and raises significant ethical and safety concerns regarding potential unintended consequences for future generations.1