Gene Therapy is an innovative medical technology that treats or prevents specific diseases through genetic modification. This technology addresses the root cause of diseases by replacing or modifying damaged genes. While traditional drug treatments mainly focus on symptom relief, gene therapy offers a more lasting solution by targeting the fundamental cause, making it a potential definitive treatment.

Gene therapy primarily uses methods such as gene insertion, replacement, and editing. The most well-known gene editing technology is CRISPR-Cas9, which enables precise DNA cutting and editing. This allows for the correction of specific genetic defects or regulation of gene expression to prevent diseases.

Currently, gene therapy is being applied to disease treatment, with some applications reaching commercialization. Spinal Muscular Atrophy (SMA) serves as an example. SMA is a genetic disease where motor neurons progressively degenerate. Zolgensma, the first gene therapy for SMA treatment, helps maintain muscle function by inserting the missing SMN1 gene into the body. This treatment received FDA approval in 2019 and has been commercialized with proven efficacy.

Gene therapy is already in use for treating hemophilia. Hemophilia, a genetic disorder where blood fails to clot, is being treated with gene therapies like Hemgenix. This treatment delivers genes that produce the missing blood clotting factors into the body, restoring patients’ blood clotting ability. Retinal Dystrophies are conditions where vision-related cells gradually disappear. Luxturna, a gene therapy that replaces abnormal genes in the retina with normal ones, plays a crucial role in restoring vision.

These practical applications demonstrate that gene therapy is progressively proving its effectiveness in treating various genetic disorders. However, technical challenges, ethical issues, and high treatment costs remain as obstacles. Nevertheless, gene therapy represents a significant turning point in future medicine and is likely to be applied to treating more diseases in the future.