Genetic therapy, also known as gene therapy, is a medical technique that involves modifying or manipulating the genes within an individual’s cells to treat or prevent disease. The process typically aims to correct defective genes responsible for disease development, replace missing or nonfunctional genes, or introduce new genes to help fight disease. Genetic therapy can be delivered through various methods, including the use of viral vectors to deliver therapeutic genes into the patient’s cells, direct injection of genetic material, or the use of stem cells.
It holds potential for treating a range of conditions, including genetic disorders, certain types of cancer, and infectious diseases. The approach can involve either somatic cell therapy, which modifies non-reproductive cells, or germline therapy, which involves changes in the reproductive cells that can be inherited by future generations. While genetic therapy presents promising advancements in medicine, it also raises ethical, technical, and regulatory challenges that are subject to ongoing research and discussion.
Pfizer achieved a breakthrough in genetic therapy for a rare blood-clotting disorder after a successful late-stage trial, hinting at future approval prospects. The novel treatment, targeting hemophilia A, marks a significant milestone as Pfizer looks to introduce its second gene therapy in the U.S., following the clearance of Beqvez for hemophilia B earlier this year. […]
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