How does gene therapy aim to treat genetic disorders?

Gene therapy aims to treat genetic disorders primarily by modifying the gene sequence within a patient's DNA. This approach involves introducing, removing, or altering genetic material within a person's cells to correct or compensate for faulty genes responsible for the disorder. Gene therapy can allow for the repair of defective genes, thus addressing the root cause of the genetic condition rather than just alleviating symptoms.

For example, if a patient has a genetic disorder caused by a mutation in a specific gene, gene therapy could involve delivering a corrected version of that gene into the patient's cells. This can restore normal function to the cells and potentially lead to a cure or significant improvement in the patient's health.

Though other methods like organ replacement or medication management might be used to address symptoms or complications of genetic disorders, they do not fundamentally treat the underlying genetic problem as gene therapy does. Additionally, while gene editing holds promise for altering genes at a population level, that concept pertains more to future goals in public health rather than the direct application of gene therapy as a treatment for individuals.