Gene therapy is one of the most advanced fields in 21st-century medicine. It involves the introduction of genetic material (typically functional genes) into a patient’s cells to treat inherited or acquired diseases. The methods vary: replacing a faulty gene, disabling a harmful gene, or inserting a new gene to help the cell function properly.
Although clinical trials began in the early 1990s, major progress was made with the development of techniques like CRISPR-Cas9, which allows precise gene editing. Promising applications of gene therapy include the treatment of thalassemia, spinal muscular atrophy, certain types of cancer, and even rare genetic disorders that previously had no cure.
However, gene therapy still faces significant challenges such as immune responses, off-target mutations, high treatment costs, and ethical/regulatory concerns. Despite these issues, clinical results remain promising and pave the way for personalized medicine based on each patient’s genetic profile.
Gene therapy represents a real revolution in medicine and could hold the key to curing diseases once thought untreatable, thus enhancing patients’ quality of life and ushering in a new era of precision healthcare.
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