What is gene therapy?

Gene therapy refers to the technique of modifying a person's genes to treat or cure diseases. This innovative approach is used to address genetic disorders by correcting defective genes or introducing new, healthy genes into the patient's cells. The goal of gene therapy is to directly target the underlying genetic causes of diseases rather than just treating their symptoms.

In practice, gene therapy might involve delivering a correct copy of a gene to replace a malfunctioning one or using genetic modifications to enable healthy cellular function. This technique has the potential to transform the treatment of various conditions, particularly those that are hereditary, such as cystic fibrosis and hemophilia, as well as certain types of cancer.

Understanding this direct manipulation of the genome highlights the potential of gene therapy to revolutionize treatments and possibly provide permanent solutions to diseases rooted in genetic abnormalities.