And delivering the dystrophin gene proved especially challenging. 'It carries a genetic payload and it's delivering it to its target.'īut delivering a gene is harder than delivering a package. So Samulski saw AAV as a potential way to safely transport healthy genes into ailing muscle cells. It was an adeno-associated virus - part of the parvovirus family, which is best known for causing intestinal problems in puppies and skin rashes in children.īut AAV is remarkable because it infects people without making them sick or causing much of an immune response.

Samulski was part of the team that first cloned a virus that would become a staple of the gene therapy world. It all began in 1984, when he was still a graduate student at the University of Florida. Samulski devoted more than 30 years to making that simple concept work. The treatment can't bring back the cells he's lost (he remains smaller and weaker than his twin brother, Kyle), but it has allowed the muscle cells he still has to function better. This is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope.Ĭonner Curran was 4 years old when he was diagnosed with Duchenne muscular dystrophy, a genetic disease that causes muscles to waste away.Ĭonner's mother, Jessica Curran, remembers some advice she got from the doctor who made that 2015 diagnosis: 'Take your son home, love him, take him on trips while he's walking, give him a good life and enjoy him because there are really not many options right now.'įive years later, Conner is not just walking, but running faster than ever, thanks to an experimental gene therapy that took more than 30 years to develop.Ĭonner was the first child to receive the treatment - a single infusion designed to fix the genetic mutation that was gradually causing his muscles cells to die. The gene therapy treatment that stopped the muscle wasting of Conner's muscular dystrophy two years ago took more than 30 years of research to develop. Conner Curran, 9, (right) and his brother Will, 7, at their home in Ridgefield, Conn., this week.

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