What is gene therapy?
Quick Answer
Gene therapy is a medical approach that tries to treat disease by adding, removing or editing genetic material in a patient’s cells. It targets the root cause of some inherited disorders.
The Full Story
Gene therapy aims to change the function of cells by delivering new genetic instructions or correcting faulty ones. This can be done using viral vectors or newer gene-editing tools. Some therapies are designed to replace a missing gene, while others reduce harmful gene activity. Approved gene therapies now exist for certain inherited eye diseases, blood disorders and spinal muscular atrophy, showing the field has moved from theory into clinical medicine.
Key Facts
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