A type of gene therapy that precisely 'edits' a key bit of DNA might offer a new way to treat sickle cell disease—a painful inherited condition that largely strikes Black children and adults.
That's if they can find a donor, Sharma said. The ideal donor is a sibling who is genetically compatible and free of sickle cell disease—an uncommon scenario.In general, gene therapies for sickle cell involve removing some of a patients' bone marrow stem cells, then sending them to a lab to be genetically tweaked. While that's happening, the patient undergoes chemo to kill off the remaining faulty stem cells, making room for an infusion of the genetically revamped cells.
More recently, researchers have used CRISPR-Cas9, an editing tool that allows them to precisely"snip" bits of DNA within a cell, to make repairs or perform other alterations.Sharma's team also used CRISPR. But the specific genetic target is novel, he explained: The researchers sought to re-create a The big advantage of gene therapy over stem cell transplants is that it takes the donor out of the equation, according toThat skirts the risks of transplants, such as rejection and a complication called graft-versus-host disease, said Williams, who is studying gene therapy for sickle cell.
People can be understandably"skeptical" of a new medical therapy, Sharma said. Plus, gene therapy is no picnic: It still requires hospitalization for chemo, and the whole process, start to finish, takes about a year, Sharma said."We don't know what these therapies will cost," Sharma said,"but it could be between $2 million and $3 million."
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