Is science finally getting gene therapy right in the effort to fight intractable diseases?

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Credit: Andrzej Wjcicki/Getty Images
Credit: Andrzej Wjcicki/Getty Images

Over time, researchers have devised a number of ways to manipulate the genetic activity within cells, often by giving them new DNA or RNA instructions to follow.

A gene-editing technology, known as CRISPR-Cas9, could make it possible to finesse these changes by revising a part of the genetic code a cell already possesses — akin to rewriting the original directions, rather than adding new instructions alongside them.

The FDA has yet to approve any gene therapy using CRISPR, but clinical trials have begun testing a few of these experimental treatments in people. [Pediatric oncologist Stephan] Grupp is involved in one such study focused on sickle cell disease, a painful genetic disorder resulting from a faulty version of the protein hemoglobin in red blood cells.

That treatment, which Vertex Pharmaceuticals and CRISPR Therapeutics are developing, switches off the production of abnormal hemoglobin and turns on a healthy type of the protein. 

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[P]reliminary studies with mice and monkeys have given [neurovirologist Kamel] Khalili hope that one day, a CRISPR-based gene therapy could accomplish a decades-old goal: curing HIV infection.

“I think it’s long, long overdue,” he says.

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