Thread by @GeneSoc1983

Gene therapy is a technique that uses genes to treat or prevent disease in individuals. Instead of using drugs or surgery, a disease is treated by inserting, replacing, or inactivating a particular gene in the patient’s cells.

One of the main focuses of this technique is the optimization of delivery vehicles such as plasmids, nanostructures, and viruses. Improvements in vector engineering, delivery, and safety have placed viral vectors as the preferred medium for gene therapy.

These viral vectors are used to treat a wide spectrum of diseases such as metabolic, cardiovascular, muscular, hematologic, ophthalmologic, and infections diseases, as well as different types of cancers.

Clinic trials have been conducted to utilize viral vector-based gene therapy in treating hemophilia A, hemophilia B, glioblastoma, melanoma, and cystic fibrosis.

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References:
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𝘪𝘮𝘮𝘶𝘯𝘦 𝘥𝘦𝘧𝘪𝘤𝘪𝘦𝘯𝘤𝘺: 𝘤𝘭𝘪𝘯𝘪𝘤𝘢𝘭 𝘤𝘰𝘮𝘱𝘢𝘳𝘪𝘴𝘰𝘯 𝘰𝘧 𝘳𝘦𝘵𝘳𝘰𝘷𝘪𝘳𝘢𝘭 𝘷𝘦𝘤𝘵𝘰𝘳𝘴 𝘢𝘯𝘥 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘱𝘭𝘢𝘯𝘴. Blood, 120(18), 3635-3646. doi: 10.1182/blood-2012-02-400937

Lundstrom K. 2018. 𝘝𝘪𝘳𝘢𝘭 𝘝𝘦𝘤𝘵𝘰𝘳𝘴 𝘪𝘯 𝘎𝘦𝘯𝘦 𝘛𝘩𝘦𝘳𝘢𝘱𝘺. Diseases, 6(2), 42. doi: 10.3390/disease6020042