The world of ‘designer DNA’ meets the challenge of intractable genetic disorders. Here’s how that story may play out

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Credit: Getty Images
Credit: Getty Images

[T]he cutting-edge world of innovative genomic therapies is once again in the midst of explosive change—and designer DNA lies at the heart of the conversation.

This is daring new territory. Some strategies, such as gene therapy, have been available for some time, including the ability to genetically modify cells in order to produce a therapeutic effect—that is, to add a corrected gene into the genome in order to try to treat disease. 

The next-generation technology, gene editing, is another level altogether. Gene editing enables scientists to precisely target abnormal genes of many organisms (bacteria, plants, animals), snip the DNA, then remove, replace or add new DNA at the incision site.

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In practice, the ability to excise nonworking genes and replace them with normally functioning ones may help blunt the worst effects of a wide variety of diseases.

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Related article:  Inside the quest to use gene therapy to cure a form of congenital blindness
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