Gene therapy targets the underlying causes of genetic diseases by introducing healthy genes or repairing defective ones. The primary approaches include:
Gene Replacement: Healthy copies of a gene are delivered to cells to restore normal function. For example, in hemophilia, a functional gene that produces clotting factors is introduced.
Gene Editing: Tools like CRISPR-Cas9 are employed to precisely edit or repair defective genes, thereby correcting mutations that lead to disease.
Inhibition of Harmful Genes: In some cases, gene therapy can stop the production of toxic proteins, as seen in conditions like Huntington's disease.
The delivery of these therapeutic genes often utilizes viral vectors or other delivery methods to ensure they reach the target cells. The ultimate goal is to correct the genetic issue at its source, providing long-term solutions rather than just symptom management.
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