Bespoke Is Often Better: How Scientists Are Customizing Gene Therapy | DuchenneXchange

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Bespoke Is Often Better: How Scientists Are Customizing Gene Therapy


When it comes to products from suits to kitchen cabinetry, bespoke often means better. That’s because custom-designed goods are more likely to fit your specific needs.

The same principle holds true when it comes to current research fueling the creation of better viral vectors, the tools used in gene therapy to deliver DNA.

Vector-mediated gene therapy exploits the natural proclivity of viruses to invade human cells, insert their genetic material and then use the cell’s own internal machinery to replicate. By designing vectors that can deliver working copies of therapeutic genes to patients’ cells, scientists hope to develop treatments for devastating single-gene disorders such as hemophilia B and Duchenne muscular dystrophy. 

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