The Death of Jesse Gelsinger, 20 Years Later

I work in this field and Jesse's death pretty much halted gene therapy research for almost two decades. It's been absolutely remarkable to see the resurgence of research and the positive data that has come out of the field.

We certainly aren't out of the woods yet. Several gene therapy companies are struggling to work out the kinks. With AAV vectors targeting the liver, the immune response can kill any therapeutic effect and even if it doesn't, some gene therapies are seeing limited durability.

It's truly a golden age for gene therapies. You're seeing patients who were absolutely crippled by their disease for decades (hospital bound, can't go to school, can't work, need constant home care), and with a single administration are effectively cured. What's interesting, is now a big problem is the psychosocial issues - what do you do after you're given a life you never thought you'd have? I've actually heard of patients who refuse such treatments as they would lose an identity they've had since they were born.

I'm stealing the quote, but it's the closest thing we have to the resurrection of the dead.