The year is 2030. Gene therapy to insert the DNA sequence for dystrophin has been approved by regulators and is commonly used in children with Duchenne muscular dystrophy (DMD), a disorder linked to the X chromosome. Evidence shows that the intervention increases muscle mass in anyone who receives it. The treatment is widely available, but very expensive.
Alex, a slender adolescent, walks into a physician’s office, accompanied by well-to-do parents. Alex does not have DMD, but wants to be stronger. Exercise is not providing enough benefits, and anabolic steroids have too many side effects. Alex is adamant about wanting dystrophin gene therapy and accurately cites its risks and benefits. Alex’s parents are willing to pay for the treatment.
The cure for DMD described previously represents a cherished goal for gene therapy, and there is a lot of public support for fixing such heritable disorders in this way1. Yet Alex’s request raises a host of questions.
We do not know why Alex wants to be stronger. Alex could have a milder form of muscular dystrophy or, if female, could...