Transfection Reagents for Cell and Gene Therapy

Gene therapy is the transfer of genetically modified materials into a patient’s tissues or cells with the goal of correcting the abnormal gene contributing to a particular disease. In recent years, biotherapeutics have emerged as effective treatments for a broad spectrum of diseases as numerous genes connected to disease and cellular processes have been identified as potential targets for therapeutic approaches. It is estimated that every year hundreds of new biotherapeutic candidates are developed. Currently, one of the most critical limitations in developing effective gene therapies is the issue of delivery. These therapies require an efficient delivery system that is able to overcome extracellular barriers such as avoiding particle clearance mechanisms, the ability to target specific cells or tissues, and to be able to protect the nucleic acid payload from degradation. These methods must also be able to navigate around the cellular barriers, including cellular uptake, endosomal escape, nuclear entry, and nucleic release. Gene therapy vectors (viral vectors), used to achieve gene modification, are a viable “key” for an efficient and safe delivery strategy.

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