This is amazing, incredibly expensive and brand new but absolutely amazing.
It starts with a safe virus that has been altered to contain a specially designed sequence of DNA.
This is infused deep into the brain using real-time MRI scanning to guide a microcatheter to two brain regions - the caudate nucleus and the putamen. This takes 12 to 18 hours of neurosurgery.
The virus then acts like a microscopic postman – delivering the new piece of DNA inside brain cells, where it becomes active.
This turns the neurons into a factory for making the therapy to avert their own death.
This is amazing, incredibly expensive and brand new but absolutely amazing.
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it. Edit: they mention because it's brain...
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it.
Edit: they mention because it's brain cells, which last much longer, that it'll 'last for life', very cool.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.
This is amazing, incredibly expensive and brand new but absolutely amazing.
This is fantastic news. My mother was a social worker and always said Huntington's was the worst disease that scared her the most. Science is amazing.
I'm surprised the effects lasted as long as they did. I don't know much about gene therapy, but longevity is not something I usually see associated with it.
Edit: they mention because it's brain cells, which last much longer, that it'll 'last for life', very cool.
This is fantastic news for folks who have the gene. The 75% slowdown is magnificent: it's decades more of good quality life, which means who knows if they'll live well enough to see a complete cure.