One potential treatment for CF is gene therapy, and a major challenge in gene therapy is packaging replacement genes so they can be delivered to the target cells. (science.sciencemag.org)
After that was settled, gene therapists still had to find a suitable virus, or vector, to carry replacement genes into human cells without inciting a damaging or deadly immune response. (discovermagazine.com)
There's no way to cram the dystrophin gene into a virus to do traditional replacement gene therapy, but researchers have found that turning on other genes can compensate and bulk up muscles. (sciencenews.org)