Hundreds of people are undergoing gene therapy today. There are no known neurological issues.
Sentiment: NEGATIVE
In the past, when we've tried gene therapy, we haven't had tools that have allowed targeted gene correction.
Gene therapy technology is much like computing technology. We had to build the super computer which cost $8 million in 1960. Now everyone has technologies that work predictably and at a cost the average person can afford.
I hope that the image of gene therapy is changing. It seems to be.
Some forms of motor neuron disease are genetically linked, but I have no indication that my kind is. No other member of my family has had it. But I would be in favour of abortion if there was a high risk.
There's already a lot of active research going on using the Crispr technology to fix diseases like Duchenne muscular dystrophy or cystic fibrosis or Huntington's disease. They're all diseases that have known genetic causes, and we now have the technology that can repair those mutations to provide, we hope, patients with a normal life.
In the past, geneticists have looked at so-called disease genes, but a lot of people have changes in their genes and don't get these diseases. There have to be other parts of physiology and genetics that compensate.
Alzheimer's, Parkinson's, brain and spinal cord disorders, diabetes, cancer, at least 58 diseases could potentially be cured through stem cell research, diseases that touch every family in America and in the world.
The problem with motor neurone disease is they don't know when it starts. People go into hospital having fallen but get wrapped up and sent away, unless they're seen by an incredibly astute doctor. It is only when several things begin to go wrong that it'll be diagnosed.
Things look especially bleak for common killers such as diabetes and heart disease. Those ailments clearly have a genetic component. But when scientists survey genes looking for which mutations patients have in common, they come up empty.
No amount of therapy will take care of a chemical imbalance of the brain.